Clinical Trials
- A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)
- A Study to Assess the Usability of the Embrace Seizure Detection Watch in Children and Young Adults With Dravet Syndrome
- A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
- A Two-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥2 Years Old and Young Adults With Dravet Syndrome
- An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
- An Open-Label Study to Investigate the Safety of Single Ascending Doses in Children and Adolescents With Dravet Syndrome
- Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)
- Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome
- Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
- Compassionate Use of Stiripentol in Dravet Syndrome
- Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies
- Fenfluramine for Adult Dravet Patients
- GABA Biomarkers in Dravet Syndrome
- Genetic Analysis Between Charlotte’s Web Responders Versus Non- Responders in a Dravet Population
- GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome
- GWPCARE5 – An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet or Lennox-Gastaut Syndromes
- Natural History Study of Infants and Children With SCN1A-positive Dravet Syndrome
- Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome
- Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome
- Stiripentol in Dravet Syndrome
- Study of Safety and Pharmacokinetics of Oral Doses of EPX-100 in Healthy Subjects.
- The Effects of Cannabidiol (CBD) on Electrical and Autonomic Cardiac Function in Children With Severe Epilepsy
- The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
- Transcranial Magnetic Stimulation to Measure Cortical Excitability in Dravet Syndrome
- Treatment of Dravet Syndrome With Fenfluramine (Expanded Access Protocol)
- Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome
- Verapamil as Therapy for Children and Young Adults With Dravet Syndrome
- ZX008 Expanded Access Protocol
- (-)- Epicatechin Becker Muscular Dystrophy
- 2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children
- A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With DMD
- A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications
- A Device for Rapid, Painless, Bedside Muscle Evaluation of Children
- A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD)
- A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD)
- A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy
- A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)
- A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
- A New Gait Assessment Scale for Duchenne Muscular Dystrophy: Test Development, Validity and Reliability
- A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
- A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
- A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
- A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
- A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
- A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
- A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
- A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
- A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
- A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
- A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
- A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
- A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
- A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)
- A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
- A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
- A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy
- A Study of Tadalafil for Duchenne Muscular Dystrophy
- A Study of TAS-205 for Duchenne Muscular Dystrophy
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
- A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
- A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
- A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
- A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
- A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
- A Study to Assess Vamorolone in Boys Ages 2 to
- A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
- A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)
- A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy
- A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to
- A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
- A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
- A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
- A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
- AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
- Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
- Aerobic Exercise in Duchenne Muscular Dystrophy
- AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD
- AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
- Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
- An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
- An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
- An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
- An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
- An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
- An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
- An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
- An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
- Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
- Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
- Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy – A Natural History Study to Predict Efficacy of Exon Skipping
- Bicycle Ergometer Training in Duchenne Muscular Dystrophy
- Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy
- Bisoprolol in DMD Early Cardiomyopathy
- Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
- Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
- Cardiomyopathy in DMD: Lisinopril vs. Losartan
- Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
- Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
- Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
- Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
- Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
- Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
- Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
- CoQ10 and Prednisone in Non-Ambulatory DMD
- Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy
- CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
- Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
- Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment
- CureDuchenne Link™: A Resource for Research
- DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
- Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
- Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy
- Digestive Events in Duchenne Muscular Dystrophy Patients
- Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
- Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
- Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy
- Dual Task in Duchenne Muscular Dystrophy
- Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life.
- Duchenne Muscular Dystrophy Clinical Trial
- Duchenne Muscular Dystrophy Heart Study
- Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
- Duchenne Muscular Dystrophy Video Assessment Registry
- E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home”
- Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
- Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients
- Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy
- Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy
- Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker.
- Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy
- Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
- Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
- Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
- Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
- Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy – Phase 1-2
- Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy
- Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
- Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
- Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
- Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy
- Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
- Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
- Evaluation of Home Based Assessments on Participants With DMD
- Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy
- Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
- Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
- Exploratory Study of NS-065/NCNP-01 in DMD
- Exploratory Study of NS-089/NCNP-02 in DMD
- Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
- Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
- Extension Study of Drisapersen in DMD Subjects
- Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
- Extension Study of NS-089/NCNP-02 in DMD
- Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
- Finding the Optimum Regimen for Duchenne Muscular Dystrophy
- Follow-up Study on Female Carriers With DMD Gene Variants
- Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
- Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
- Gentamicin Treatment of Muscular Dystrophy
- Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability Study
- Heart Rate Variability in Duchenne Muscular Dystrophy During Computer Task
- Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
- High-dose Prednisone in Duchenne Muscular Dystrophy
- HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
- HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
- Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
- Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy
- Investigation of the Validity and Reliability of the Kinesthetic and Visual Imagery Questionnaire
- Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption
- KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
- L-citrulline and Metformin in Duchenne’s Muscular Dystrophy
- Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
- Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
- Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
- Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
- Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance
- Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle
- Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
- Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
- Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
- Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study
- Natural History of Duchenne Muscular Dystrophy
- Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)
- Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
- Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Its Effects on Motor Functions
- Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
- Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
- Once Weekly Infant Corticosteroid Trial for DMD
- Open Label Extension Study of HT-100 in Patients With DMD
- Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
- Open-label Extension of the HOPE-2 Trial
- Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
- Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study
- PDE Inhibitors in DMD Study (Acute Dosing Study)
- Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy
- Pediatric Radio Frequency Coils Generic
- Pentoxifylline in Duchenne Muscular Dystrophy
- Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
- Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
- Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy
- Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
- Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
- Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
- Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
- Phase I/II Study of SRP-4053 in DMD Patients
- Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy
- Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
- Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
- Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
- Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
- Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
- Physical Activity Level and Cognitive Functions in Children With Duchenne Muscular Dystrophy
- Physical Activity Level of Norwegian Boys With Duchenne Muscular Dystrophy
- Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval
- Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
- Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device
- Prognostic Factors Affecting Duchenne Muscular Dystrophy
- Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
- Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy
- Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)
- Ramipril Versus Carvedilol in Duchenne and Becker Patients
- Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy
- Relationship Between PFTs and Pdi in DMD
- Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy
- Research of Biomarkers in Duchenne Muscular Dystrophy Patients
- Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
- Rimeporide in Patients With Duchenne Muscular Dystrophy
- Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
- Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
- Safety and Efficacy of P-188 NF in DMD Patients
- Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
- Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
- Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
- Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
- Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
- Safety Study of BLS-M22 in Healthy Volunteers
- Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
- Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
- Safety Study of Flavocoxid in Duchenne Muscular Dystrophy
- Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
- Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
- Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
- Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy
- Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
- Sleep Intervention in Young Boys With Duchenne Muscular Dystrophy
- Sodium Nitrate to Improve Blood Flow
- Spironolactone Versus Prednisolone in DMD
- Stacking Exercises Aid the Decline in FVC and Sick Time
- Stem Cell Therapy in Duchenne Muscular Dystrophy
- Strength Training in Duchenne Muscular Dystrophy
- Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
- Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
- Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
- Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
- Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
- Study of DS-5141b in Participants With Duchenne Muscular Dystrophy
- Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
- Study of Eteplirsen in DMD Patients
- Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
- Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
- Study of SRP-4045 and SRP-4053 in DMD Patients
- Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
- Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.
- Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
- Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
- Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD
- Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
- Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy
- Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
- Tadalafil as Adjuvant Therapy for DMD
- Tadalafil in Becker Muscular Dystrophy
- Tamoxifen in Duchenne Muscular Dystrophy
- Telerehabilitation in Duchenne Muscular Dystrophy
- Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne’s Muscular Dystrophy
- Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
- The Burden of Access in Duchenne Muscular Dystrophy in the US
- The Duchenne Registry
- The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy
- The Effect of Telerehabilitation of Patients With Duchenne Muscular Dystrophy
- The Effects of Trunk Exercises on Upper Extremity and Respiratory Functions in DMD
- The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD
- The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy
- The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
- The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
- The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
- The Relationship Between Foot Posture and Performance and Ambulation in Patients With Duchenne Muscular Dystrophy
- The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD
- The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
- Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
- This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
- To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
- Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients
- Treatment Effect of Tamoxifen on Patients With DMD
- Treatment of a Single Patient With CRD-TMH-001
- Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
- Trunk Oriented Exercises Versus Whole-body Vibration for Duchenne Muscular Dystrophy
- Upper Limb Assessment in Duchenne Muscular Dystrophy
- Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
- Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD
- User-centred Assistive System for Arm Functions in Neuromuscular Subjects
- Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
- Virtual Reality in Individuals With Duchenne Muscular Dystrophy
- Weekend Steroids and Exercise as Therapy for DMD
- Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy
- Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
- A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature
- A Study of PEG-somatropin in the Treatment of Children With Idiopathic Short Stature
- A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation
- Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
- Clinical Care Consortium of Telomere-Associated Ailments (CCCTAA) Database
- Haploidentical HCT for Severe Aplastic Anemia
- Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
- Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)
- Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
- Artificial Intelligence-assisted Evaluation of Pigmented Skin Lesions
- Atypical MOLes and Melanoma Early Detection Study (MoleMed)
- BioMEL- Diagnostic and Prognostic Factors in Melanoma.
- Evaluation of 20% Betulinic Acid Ointment for Treatment of Dysplastic Nevi (Moderate to Severe Dysplasia)
- Internet-based Intervention for Skin Self-Examination in Participants With Increased Risk for Melanoma
- Prospective Study of 2 mm Margins for the Biopsy of Dysplastic Nevi
- A Comparative Study of the Healing of Chronic Ulcers of Recessive Epidermolysis Bullosa : Dressing vs Amniotic Membrane
- A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial
- A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
- A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
- A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa
- A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa
- A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa
- A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
- A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
- A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
- Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)
- Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
- Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
- Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Biochemical Correction of Severe EB by Allo HSCT and “Off-the-shelf” MSCs
- Characteristics of Adult Patients With Recessive Dystrophic Epidermolysis Bullosa
- Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa
- Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.
- Dose-ranging Study of Dentoxol® Mouthrinse for Managing Oral Symptoms in People With Epidermolysis Bullosa.
- EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
- ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
- Evaluation of the Efficacy of ROPIVACAINE in Children and Young Adults With Hereditary Epidermolysis Bullosa
- Extension Study to PTR-01-002
- Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
- Gentamicin for RDEB
- Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
- Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes
- Growth Hormone in EB
- Gynecological Follow-up of Patients With Dystrophic Epidermolysis Bullosa (EBD)
- Improve Adherence to Weak or Strong Opioid Analgesics at the Time of Care in Children With Hereditary Epidermolysis Bullosa
- Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Long-Term Follow-up Protocol
- Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
- Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
- MSC EVs in Dystrophic Epidermolysis Bullosa
- MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
- Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients
- Observational Study of a Cohort of Patients With Hereditary Epidermolysis Bullosa
- Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10)
- Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa
- Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
- Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa
- Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa
- Pregabalin Treatment for RDEB Pain and Itch
- Proof of Concept Study for a Dressing Glove
- Prospective, Longitudinal Natural History Study in Dystrophic Epidermolysis Bullosa
- Randomised Double Blind Crossover Placebo Controlled Study to Evaluate the Efficacy of Tetracycline in Epidermolysis Bullosa
- Randomised Double Blind Placebo Controlled Cross Over Design of the Efficacy of Topical Morphine for Inflammatory Pain in Children With Epidermolysis Bullosa
- Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
- Rigosertib for RDEB-SCC
- Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC
- Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB
- Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa
- Short Term Observational Study in DEB Patients
- Study of Alwextin® Cream in Treating Epidermolysis Bullosa
- Study of Cellutome System for Treatment of Individual Lesions in EB Pts
- Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
- Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
- Study of the Nutritional, Metabolic, and Body Composition Profile in Children and Adolescents With Epidermolysis Bullosa
- Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
- Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa
- Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
- The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial
- The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa
- The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
- The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB).
- Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa
- Topical BPM31510 3.0% Cream in Patients With Epidermolysis Bullosa
- Topical Gentamicin Nonsense Suppression Therapy of EB
- Topical QR-313 in Dominant Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene
- Treatment of Chronic and Non-Chronic Wounds in Patients With Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard of Care
- Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)
- Trial To Assess Efficacy Of A Chimeric Skin In Patients With Epidermolysys Bullosa
- Uses of Irradiated Human Amniotic Membrane in the Treatment of Dystrophic Epidermolysis Bullosa Patients
- Activin A and Inhibin A in Predicting Outcome of Pregnancies of Unknown Location After Assisted Reproductive Technology
- Adnexal Mass After Methotrexate Treatment for Ectopic Pregnancies
- Are Serum Levels of Vascular Endothelial Growth Factor a Marker for the Early Diagnosis of Ectopic Pregnancy?
- Assessment of Biomarkers in Ectopic Pregnancy
- Assessment of Biomarkers in Pregnancy on Unknown Location and Ectopic Pregnancy
- BHCG Level in Day 4,7, in Comparison to Day 10 as an Indicator for Treatment Success
- Comparative Study on the Efficacy of Ovarian Stimulation Protocols on the Success Rate of ICSI in Female Infertility
- Comparison of Two Different Approaches in the Surgical Treatment of Tubal Ectopic Pregnancy
- Cornual Wedge Resection Outcome
- Ectopic Pregnancy Biomarkers
- Effectiveness Of Expectant Management In The Tubal Ectopic Pregnancy
- Evaluation of Different Doses of Letrozole in Ectopic Pregnancy
- Evaluation of Ipsilateral Tubal Patency After Conservative Medical or Surgical Treatment.
- Evaluation of the Axonemal Dynein Heavy Chain 5 and Creatine Kinase Concentration in Cervical Fluid for Early Detection of the Ectopic Pregnancy
- Expectant Management of Ectopic Pregnancy
- Hysteroscopic Metroplasty in Unexplained Infertile Women With Dysmorphic Uterus
- Improving Mental Health Following Early PREgnancy Loss Using a Brief Cognitive Task
- Kisspeptin Levels in Early Pregnancy
- Laparoscopic Tubal Preservation Surgery
- Medical Treatment in Ectopic Tubal Pregnancy
- Methotrexate Treatment for Ectopic Pregnancy
- Optimal Treatment for Women With a Persisting Pregnancy of Unknown Location
- Pregnancies of Uncertain Location or Viability Research
- Psychological Impact of Pregnancy of Unknown Location
- Randomized Population-Based Study on Chlamydia Trachomatis Screening
- RCT for Women With a Persisting Pregnancy of Unknown Location
- Relationship Between Ectopic Pregnancy and Thyroid Disorders
- Reproductive Outcomes After a Previous Episode of Tubal Ectopic Pregnancy in Patients Managed Expectantly and Surgically
- Risk Factors of Ectopic Pregnancy
- Risk of Ectopic Pregnancy and Variation of Luteal Estradiol and Progesterone Levels in IVF
- Sonographic Assessment and Visualization of Ectopics in Emergency Medicine
- The Aromatase Inhibitor and Gnrh Antagonist Versus Methotrexate for Management of Undisturbed Ectopic Pregnancy
- The Correlation Between Uterine Endometrial Pattern and Ectopic Pregnancy
- The Impact of Salpingectomy and Single Dose Systemic Methotrexate Treatments on Ovarian Reserve in Ectopic Pregnancy
- Two-Dose Methotrexate for Ectopic Pregnancy
- Vitamin D in Ectopic Pregnancies