Clinical Trials
- A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
- A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
- An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
- An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
- Individual Patient Compassionate Use of Mepsevii
- Longitudinal Studies of Brain Structure and Function in MPS Disorders
- Mucopolysaccharidosis VII Disease Monitoring Program
- Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
- A Phase II Trial of Ipilimumab and Nivolumab for the Treatment of Rare Cancers
- A Randomized Phase II Open Label Study to Compare the Safety and Efficacy of Subcutaneous Dalteparin Versus Direct Oral Anticoagulants for Cancer-associated Venous Thromboembolism
- A Retrospective Cohort Study for Main Postoperative Complications After SARS-CoV-2 Infection
- Antineoplaston Therapy in Treating Patients With Cancer of the Small Intestine
- Assessment of a Serious Illness Conversation Guide (SICG) in Advanced Gastro-Intestinal Cancers
- Canady Helios Cold Plasma Scalpel Treatment at the Surgical Margin and Macroscopic Tumor Sites
- Capecitabine and Oxaliplatin in Adenocarcinoma of the Small Bowel and Ampulla of Vater
- CAPOX in KRAS Wild-Type Advanced Adenocarcinoma of the Small Bowel or Ampulla of Vater
- Colonization of Bile Ducts and Postoperative Infectious Complications of Pancreaticoduodenectomies
- Combination Chemotherapy in Treating Patients With Stage III Ovarian Epithelial Cancer or Gastrointestinal Cancer
- Combination Chemotherapy Plus Interferon Alfa Followed by Filgrastim in Treating Patients With Gastrointestinal Tract Cancer
- Combination of Brivanib With 5-Fluorouracil/Leucovorin (5FU/LV) and 5-Fluorouracil/Leucovorin/Irinotecan (FOLFIRI)
- Data Collection of PillCamTM SB3 Capsule Endoscopy and Device-Assisted Enteroscopy Routine Care Procedures (GPS Study)
- Diagnostic Performance Indicators in Upper GI Endoscopy:PROSPERO Study
- Duodenal Cancer – Retrospective Analysis
- Effectiveness of Low Dose Aspirin in Decreasing the Chance Getting Stomach and Intestine Cancer
- Efficacy of 2-Liter Mixed Preparation With Bisacodyl Plus Polyethylene Glycol and 4-Liter Polyethylene Glycol for Colon Cleansing in Patients With Prior History of Colorectal Resection
- Endoscopic Evaluation of Duodenal Polyposis in Patients With Familial Adenomatous Polyposis (FAP)
- Endoscopic Placement of Metal Stent in Patients With Cancer-Related Bowel Obstruction
- Endoscopic Placement of Metal Stents in Treating Patients With Cancer- Related Duodenal Obstruction
- Enteroscopy for Early Diagnosis of Tumors in Celiac Disease
- Epoetin Alfa in Treating Anemia in Patients Who Are Receiving Chemotherapy
- Exisulind in Preventing Polyps in Patients With Familial Adenomatous Polyposis
- Gemcitabine and Radiation Therapy in Treating Patients With Locally Advanced Upper Gastrointestinal Cancer
- Gemcitabine Hydrochloride, Oxaliplatin, and Erlotinib Hydrochloride in Treating Patients With Advanced Biliary Tract Cancer, Pancreatic Cancer, Duodenal Cancer, or Ampullary Cancer
- Gemcitabine, Docetaxel, and Filgrastim in Treating Patients With Recurrent or Persistent Leiomyosarcoma or Soft Tissue Sarcoma
- GI Organ Tracking Via Balloon Applicators
- HFNO Combined With NPA Reduces Hypoxia During Sedated Gastrointestinal Endoscopy In Obese Patients
- HFNO Reduces Hypoxia During Sedated Gastroscopy or Colonoscopy in High Risk Patients
- Improving Cancer Symptom Management
- Interferon Alfa-2b in Treating Patients With Advanced Low-Grade Non-Hodgkin’s Lymphoma
- Interventional Endoscopy Database for Pancreatico-biliary, Gastrointestinal and Esophageal Disorders
- Irinotecan, Fluorouracil, and Leucovorin in Treating Patients With Advanced Gastrointestinal Cancer
- Irinotecan, Oxaliplatin, and Capecitabine as First-Line Therapy in Treating Patients With Metastatic or Unresectable Locally Advanced Small Bowel Cancer
- JAB-21822 in Combination With Cetuximab in Patients With Advanced CRC and Other Solid Tumors With KRAS G12C Mutation
- Long-term Outcomes After Resection of Primary Duodenal Adenocarcinoma
- Main Postoperative Complications After COVID-19
- Monitoring of patIents With Microdialysis Following Pancreaticoduodenectomy
- Nitrocamptothecin in Treating Patients With Soft Tissue Sarcomas
- Nutrition in Gastrointestinal Tumors
- Octreotide as Palliative Therapy for Cancer-Related Bowel Obstruction That Cannot Be Removed by Surgery
- Oxaliplatin Plus Capecitabine in Treating Patients With Colorectal, Appendix, or Small Bowel Cancer
- Oxaliplatin Plus Irinotecan in Treating Patients With Metastatic Gastrointestinal Cancer
- Palonosetron Hydrochloride in Preventing Nausea and Vomiting Caused by Radiation Therapy in Patients With Primary Abdominal Cancer
- Patient Reported Scale vs CTCAE Grading: A Prospective Comparator Study
- PeRsonalizing the Approach to the Oncologic Frail Individual Through Tailored Assessment and Intervention (PROFIT Study)
- Postoperative Oral Nutritional Supplementation After Major Gastrointestinal Surgery
- PreOPerative Imaging of NeuroEndocrine Tumors
- Programs To Support You During Chemotherapy
- Prospective Collection of PillCam SB3 Videos and Raw Data Files for Future Developments (SODA)
- Proximal Roux-en-y Gastrojejunal Anastomosis on Delayed Gastric Emptying After Pylorus-resecting Pancreaticoduodenectomy
- Psychological and Emotional Impact in Patients Undergoing Treatment For Metastatic Cancer Either in a Clinical Trial or as Standard Off-Trial Therapy
- Quality of Life in Patients Undergoing Radiation Therapy for Primary Lung Cancer, Head and Neck Cancer, or Gastrointestinal Cancer
- Quebec Pancreas Cancer Study
- Randomized Controlled Trial on Pancreatic Stent Tube in Pancreaticoduodenectomy
- Role of Proton Pump Inhibitors on the Postoperative Course Following Pancreaticoduodenectomy
- Sensitivity and Specificity of NGS in Detecting bMSI in Patients With Gastric, Duodenal and Small Intestinal Cancer
- sFOLFOXIRI in Advanced Gastroesophageal Cancer, (SAGE)
- Stereotactic Body Radiotherapy in Patients With Rare Oligometastatic Cancers (OligoRARE)
- Study of Irinotecan Administered as a Continuous Infusion and Radiation Therapy for Upper Gastrointestinal Cancers
- The GI Binder: A Psychoeducational Intervention for Gastrointestinal Cancer Patients
- The Impact of a Patient Education Intervention for Ambulatory Oncology Patients
- The SOAR Study: a Study of Geriatric Assessment and Exercise for Older Adults and Their Support Person
- Trial on the Evaluation of Pylorus-ring in Pancreaticoduodenectomy
- Use of the EnLightTM and LightPathTM Imaging Systems in Gastrointestinal Tumour Surgery
- Using Electrical Bioimpedance Assessments to Estimate Perioperative Total Body Water and Postoperative Fluid Need
- Vaccine Therapy in Treating Patients With Cancer of the Gastrointestinal Tract
- Virtual Reality for GI Cancer Pain to Improve Patient Reported Outcomes
- Visceral Lymphatic Mapping Using Isosulfan Blue in Patients With Cancer of the Pancreas, Colon, Stomach, Small Intestine, or Gallbladder
- Bryostatin 1 Plus Vincristine in Treating Patients With Recurrent or Refractory HIV-Related Lymphoma
- Combination Chemotherapy and Rituximab in Treating Patients With HIV-Associated Stage I, Stage II, Stage III, or Stage IV Non-Hodgkin’s Lymphoma
- Combination Chemotherapy and Rituximab in Treating Young Patients With Recurrent or Refractory Non-Hodgkin’s Lymphoma or Acute Lymphoblastic Leukemia
- Intravenous Chemotherapy or Oral Chemotherapy in Treating Patients With Previously Untreated Stage III-IV HIV-Associated Non-Hodgkin Lymphoma
- Radiolabeled Monoclonal Antibody With or Without Peripheral Stem Cell Transplantation in Treating Children With Recurrent or Refractory Lymphoma
- Rituximab, Rasburicase, and Combination Chemotherapy in Treating Young Patients With Newly Diagnosed Advanced B-Cell Leukemia or Lymphoma
- Topotecan Hydrochloride in Treating Children With Meningeal Cancer That Has Not Responded to Previous Treatment
- Vorinostat With or Without Isotretinoin in Treating Young Patients With Recurrent or Refractory Solid Tumors, Lymphoma, or Leukemia
- A Long-Term Study of Healthy Adults Vaccinated With One Dose of Smallpox Vaccine (LISTER Strain)
- A Phase 4, Observational Field Study to Evaluate TPOXX in Patients With Smallpox
- A Phase II Trial to Compare a Liquid-frozen and a Freeze-dried Formulation of IMVAMUNE (MVA-BN®) Smallpox Vaccine in Vaccinia-naïve Healthy Subjects
- A Randomized, Double-blind, Placebo-controlled Study on Immunogenicity and Safety of MVA-BN (IMVAMUNE™) Smallpox Vaccine in Healthy Subjects
- A Study of Dryvax Vaccine Against Smallpox in Previously Unvaccinated Adults
- A Study to Evaluate Safety and Immunogenicity of One and Two Doses of IMVAMUNE® Smallpox Vaccine in 56-80 Year Old Vaccinia-experienced Subjects
- A Trial to Assess the Safety, Tolerability, and Pharmacokinetics of the Anti-Orthopoxvirus Compound Tecovirimat
- A Trial to Evaluate Immunogenicity and Safety of Three Consecutive Production Lots of IMVAMUNE® (MVA-BN®) Smallpox Vaccine in Healthy, Vaccinia-naïve Subjects
- ACAM 3000 MVA at Harvard Medical School
- An Open-Label Phase II Study to Evaluate Immunogenicity and Safety of a Single IMVAMUNE Booster Vaccination Two Years After the Last IMVAMUNE Vaccination in Former POX-MVA-005 Vaccinees
- APSV in Vaccinia Naive Adults
- Assessment of Myocarditis After Replication-Deficient Smallpox Immunization
- Combination Study With MVA BN and Dryvax
- Dose Safety, Tolerability, and Immunogenicity of a New Smallpox Vaccine in Adults Without Previous Smallpox Vaccination
- Dose Study of ACAM2000 Smallpox Vaccine in Previously Vaccinated Adults
- Dose-finding Study to Evaluate Immunogenicity of Three Different Dose Levels of the IMVAMUNE (MVA-BN) Smallpox Vaccine.
- Dressing Preparations for Smallpox
- Drug-drug Interaction Study of TPOXX When Co-administered With Phosphate Binders
- Dryvax Dilution-Prev Vacc Adults
- Effect of Dose, Safety, Tolerability of a New Smallpox Vaccine in Adults Without Previous Smallpox Vaccination
- Evaluation of Take and Safety of a Smallpox Vaccine in Healthy Young Adults
- Expanded Dryvax Dilution Study in Previously Vaccinated Adults
- Freeze-Dried MVA-BN® Lot Consistency Smallpox Trial
- Human Immune Responses Smallpox
- Lyophilized IMVAMUNE® (1×10^8 TCID50) Versus Liquid IMVAMUNE® (1×10^8 TCID50) Administered Subcutaneously and a Lower Dose Liquid IMVAMUNE® (2×10^7 TCID50) Administered Intradermally
- MVA Post-Event: Administration Timing and Boost Study
- Phase 2 Study of Safety, Tolerability, and Immunogenicity of Three Dose Levels of ACAM3000 Smallpox Vaccine
- Phase I Trial of an Investigational Small Pox Medication
- Phase II Trial to Assess Safety and Immunogenicity of IMVAMUNE®
- Poly-ICLC to Prevent Respiratory Viral Infections A Safety Study
- Post-licensure Department of Defense (DOD) Screening Accuracy Study in Military Personnel
- Randomized, Open-label Phase II Trial to Assess the Safety and Immunogenicity of MVA-BN Smallpox Vaccine in Immunocompromised Subjects With HIV Infection
- Safety and Efficacy of CJ Smallpox Vaccine in Healthy Volunteers
- Safety and Efficacy of CJ Smallpox Vaccine in Previously Vaccinated Healthy Volunteers
- Safety and Efficacy of CJ-50300 in Healthy Volunteers
- Safety and Immunogenicity of CJ-50300
- Safety and Immunogenicity Study of LC16m8, a Modified Smallpox Vaccine, in Healthy, Previously Unvaccinated Volunteers
- Safety and Tolerability Study of the VAC∆6 Vaccine in Volunteers Aged 18-40 Years
- Safety Study of MVA Smallpox Vaccine in HIV-positive Subjects Who Are Vaccinia Naive
- Safety Study of MVA Smallpox Vaccine in Subjects With a History of Atopic Dermatitis (AD)
- Safety Surveillance Cohort Study of Vaccinia Vaccine (ACAM2000®)
- Safety Surveillance Study of ACAM2000® Vaccinia Vaccine
- Safety, Tolerability and Pharmacokinetics of TPOXX When Administered Orally for 28 Day
- Safety, Tolerability and PK of TPOXX in Adults Weighing More Than 120 KG
- Safety, Tolerability, and Immune Response of ACAM3000 Modified Vaccinia Ankara (MVA) Smallpox Vaccine in Adults
- Study of Reactogenicity, Safety, Immunogenicity, and Pock Lesion Formation of a Cell-Cultured Smallpox Vaccine Compared to Dryvax®
- Study on Safety, Tolerability and Immunogenicity of an MVA Vaccine Administered to Healthy Subjects
- Study to Assess the Safety and Pharmacokinetics of TPOXX® When Administered Orally for 28 Days
- Take Rate, Immunogenicity and Safety of Elstree-BN Smallpox Vaccine in Healthy Vaccinia-Naive Subjects
- Tecovirimat (ST-246) Treatment for Orthopox Virus Exposure
- Vaccinia Virus Vaccine (APSV) in Vaccinia- Naive Subjects: PILOT
- VHA Clinicians and Bioterror Events: Interactive Web-based Learning
- Capecitabine, Temozolomide and Bevacizumab for Metastatic or Unresectable Pancreatic Neuroendocrine Tumors
- Dovitinib Lactate in Treating Patients With Pancreatic Neuroendocrine Tumors
- Everolimus and Vatalanib in Treating Patients With Advanced Solid Tumors
- Everolimus in Patients With Pancreatic Neuroendocrine Tumors Metastatic to the Liver Previously Treated With Surgery
- Ga-DOTATOC Versus Octreoscan + CT
- Pharmacokinetically Guided Everolimus in Patients With Breast Cancer, Pancreatic Neuroendocrine Tumors, or Kidney Cancer
- Regorafenib in Treating Patients With Advanced or Metastatic Neuroendocrine Tumors
- Romidepsin in Treating Patients With Locally Advanced or Metastatic Neuroendocrine Tumors
- Temozolomide With or Without Capecitabine in Treating Patients With Advanced Pancreatic Neuroendocrine Tumors
- A Phase 2 Trial of Deep Brain Stimulation for Spasmodic Dysphonia
- Assessment of Laryngopharyngeal Sensation in Adductor Spasmodic Dysphonia
- Brain Networks in Dystonia
- Characteristics of Idiopathic Familial Voice Disorders
- Characterization of Clinical Phenotypes of Laryngeal Dystonia and Voice Tremor
- Cortical Silent Period From Cricothyroid Muscle in Laryngeal Dystonia Patients and Control Subjects
- DaxibotulinumtoxinA Injection for Treatment of Adductor Spasmodic Dysphonia
- Deep Brain Stimulation in Laryngeal Dystonia and Voice Tremor
- Effect of Vocal Exercise After Botulinum Toxin Injection for Spasmodic Dysphonia
- Examining the Spasmodic Dysphonia Diagnosis and Assessment Procedure (SD-DAP) for Measuring Symptom Change
- Imaging Genetics of Spasmodic Dysphonia
- Laryngeal Vibration for Spasmodic Dysphonia
- Neuropathology of Spasmodic Dysphonia
- Role of Neurotransmission and Functional CNS Networks in Spasmodic Dysphonia
- rTMS in Spasmodic Dysphonia
- Sensory Function in Idiopathic Voice Disorders
- Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor
- Spasmodic Dysphonia Pain
- Study of Air Stream in Voice Production
- Thalamic Deep Brain Stimulation for Spasmodic Dysphonia- DEBUSSY Trial
- Understanding Disorder-specific Neural Pathophysiology in Laryngeal Dystonia and Voice Tremor
- Voice Tremor in Spasmodic Dysphonia: Central Mechanisms and Treatment Response
- A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
- Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
- LYSO-PROVE – Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease
- Natural History Study of Children With Metachromatic Leukodystrophy
- Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients
- SPL Insufficiency Syndrome (SPLIS): a SPLIS Observational Study and Patient Registry (International)
- A Peer E-mentoring Intervention to Improve Employment
- A Randomized Exercise Trial for Wheelchair Users
- A Wheelchair Propulsion Training Program
- Anorectal Dysfunction in Patients Suffering From Spina Bifida : From Clinic to Neuro-epithelial Function (ANOSPIN)
- Awareness Levels of Caregivers of Disabled Children
- Children and Teens in Charge of Their Health
- CIC Behavioral Economics in Children With Spina Bifida
- Cognitive Rehabilitation in Patients With Spina Bifida
- Comparing Urinary Tract Infections in Children With Spina Bifida Using Two Types of Catheters for Catheterization
- Coping Skills Training (CST) for Children With Chronic Health Conditions
- Downstream Effects of Personalized ‘Top-down’ Participation-based Interventions Among Youth With Physical Disabilities
- Effects of Progressive Resistance Exercise Training on Endurance and Functional Mobility in Children With Spina Bifida
- Enema Device for Children With Spina Bifida
- Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders
- Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders
- Fecal Microbium Change in Pediatric Patients With Spina Bifida: Prospective Case-control Study
- Fetal Endoscopic Surgery for Spina Bifida
- Fetoscopic Open Spina Bifida Repair Using the SAFER Technique
- Fetoscopic Repair of Isolated Fetal Spina Bifida
- Grand Valley State University (GVSU) Skills on Wheels
- Improving Genetic Counseling for Patients With Spina Bifida Using Next Generation Sequencing
- In Utero Fetoscopic Repair Program for Sacral Myelomeningoceles and Mye-LDM
- In-Utero Endoscopic Correction of Spina Bifida
- Incidence of Pregnancies and Births With Spina Bifida in Denmark in 2008-2014
- Incontinence and Quality of Life in Children With Spina Bifida
- Interrater Reliability of Infant Motor Profile
- Intervention on Bone Health in Wheelchair Users
- Investigation of Parents’ Anxiety Level and Health Related Quality of Life in Different Types of Physical Disabilities
- Laparotomy Versus Percutaneous Endoscopic Correction of Myelomeningocele
- Lumbar to Sacral Ventral Nerve Re-Routing
- Medication Management Among Individuals With Neurodevelopmental Disabilities
- Metformin in Children With Motor Deficit
- Micro Ribonucleic Acid (miRNA) Markers of Hydrocephalus in Intraventricular Hemorrhage (IVH)
- Minimally Invasive Fetoscopic Regenerative Repair of Spina Bifida – A Pilot Study
- Mobile Health Self-Management and Support System for Chronic and Complex Health Conditions
- Movement-2-Music: Lakeshore Examination of Activity, Disability, and Exercise Response Study
- Nerve Rerouting Treatment for Neurogenic Bladder in Spina Bifida
- Parental Perspectives of Using Toy Cars on Social Interactions of Preschool Children With Mobility Impairments
- Post-operative Lite Run Study
- Prevention of Post Operative Bone Loss in Children
- Prospective Cohort of Transitional Urology Patients
- Psychosocial Adjustment of Adolescents With Spina Bifida
- Qualitative Assessment of the Impact of the Connected Catheter on Quality of Life and Participation
- READYorNot[TM] Brain-Based Disabilities Trial
- Retrograde Colonic Irrigation to Manage Spina Bifida Functional Digestive Sequelae: a Multicenter, Prospective, Randomized Controlled Trial
- Robotic Walking for Children Who Cannot Walk
- Sensory and Motor Proficiency For Children With Spina Bifida
- Spina Bifida in Daily Life: an Exploratory Study
- Study of Genetic Risk Factors for Spina Bifida and Anencephaly
- Telemedicine in Spina Bifida Transition: A Pilot Study
- The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
- The Impact of Community-based Wellness Programs on The Triple Aim
- The Impact of Participation on Body Functions Among Youth With Physical Disabilities
- The Relationship Between Nocturnal Enuresis And Spina Bifida Occulta
- The Role of Active Administration of Peristeen Bowel Evacuation for the Management of Urinary Tract Infection
- Urinary Markers of Detrusor Overactivity in Spina Bifida Patients
- Urological and Renal Disease Engaging Adolescents in Adherence Collaborative Trial
- Urothelium Tissue Engineering Using Biopsies From Transurethral Resection of Prostate
- Use of a Diurnal Indwelling Urethral Catheter to Improve Quality of Life
- Virtual Peer Health Coaching for Adolescents With Disabilities
- Virtual Reality Based Testing of Power Wheelchair Driving Skills
- Yulex Glove Prospective Study in Spina Bifida
- A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
- An Observational Study to Assess Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)
- Differential Study of Muscle Transcriptome
- Dutasteride to Treat Spinal and Bulbar Muscular Atrophy (SBMA)
- Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy
- High Intensity Training in Patients With Spinal and Bulbar Muscular Atrophy
- Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
- Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg “All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)”
- Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy
- Alternative Treatment of Giant Spinal Schwannomas
- Beta-Tricalcium Phosphate Bone Graft in Treating Patients Undergoing Surgery For Metastatic Spine Cancer
- Bevacizumab and Irinotecan in Treating Young Patients With Recurrent, Progressive, or Refractory Glioma, Medulloblastoma, Ependymoma, or Low Grade Glioma
- Bone Graft Materials Observational Registry
- Direct Injection of Alcohol for the Treatment of Spinal Tumors
- Effect(s) of Esketamine Combined With Pregabalin on Postsurgical Pain in Spinal Cord Neoplasms Patients
- Evaluation of Effectiveness of CyberKnife Stereotactic Radiosurgery for Spinal Tumors
- Evaluation of Single Session Stereotactic Body Radiotherapy
- FDG PET-MRI for the Diagnosis of Spinal Cord Lesions
- Minimally Invasive Surgery in Treating Patients With Spinal Tumors
- Neurosurgical Transitional Care Programme
- Robotic-Assisted Surgery in Treating Patients With Spine Tumors
- Surgical Treatment of Intra-spinal Canal Tumors Via Trans-Quadrant Channel
- The Development of a Vertebra Localizing Aid Medical Device
- Validation of Pre-clinical Nano-Based Analgesics in Cells From Human Dorsal Root Ganglia
- Vorinostat, Temozolomide, or Bevacizumab in Combination With Radiation Therapy Followed by Bevacizumab and Temozolomide in Young Patients With Newly Diagnosed High-Grade Glioma
- A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
- A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
- A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies.
- A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
- A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
- A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
- A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
- A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
- A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
- A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
- A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam
- A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulatory Children With Spinal Muscular Atrophy
- A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
- A Trial of Hydroxyurea in Spinal Muscular Atrophy
- Adults With SMA Treated With Nusinersen
- An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
- An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
- An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
- An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
- An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
- AveXis Managed Access Program Cohort for Access to AVXS-101
- Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor
- Clinical Study of Spinal Muscular Atrophy
- Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
- COMMAND Early Feasibility Study (EFS)
- Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
- Effects of Power Mobility on Young Children With Severe Motor Impairments
- Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
- Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy
- Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
- Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform.
- European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
- Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid
- Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
- Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
- Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
- Flu Vaccine Study in Neuromuscular Patients 2011
- Hippotherapy in Children With Spinal Muscular Atrophy
- Holter of Movement in Patients With SMA Undergoing Treatment.
- Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene
- Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis
- Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
- Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
- Long-term Follow-Up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
- Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
- Measuring Levels of SMN in Blood Samples of SMA Patients
- Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
- Mechanisms of Cell Death in Spinal Muscular Atrophy
- Metabolomics of Children With SMA
- Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®
- Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
- Multi-disease Carrier Screening Test Validation
- Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy
- National Registry for Egyptian Pediatric Neuromuscular Diseases
- Natural History of Types 2 and 3 SMA in Taiwan
- Neonatal Spinal Muscular Atrophy (SMA) Screening
- New Imaging Biomarkers for Muscular Diseases – Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy
- Newborn Screening for Spinal Muscular Atrophy
- Newborn Screening for Spinal Muscular Atrophy (SMA) – a Proof of Principle Study Using Anonymised Blood Spots.
- Optimum Insufflation Capacity in NMD
- Pediatric Powered Wheelchair Standing Devices: An Exploratory Study
- Personalized Medicine for SMA: a Translational Project
- Phase II Study of Leuprolide and Testosterone for Men With Kennedy’s Disease or Other Motor Neuron Disease
- Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
- Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
- Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women
- Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
- Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices
- Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments
- Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
- Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
- Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System
- Reciprocating Gait Orthoses for Paraplegia Patients
- Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
- Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy
- Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
- Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
- Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen
- Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
- Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
- Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
- Schulze Muscular Dystrophy Ability Clinical Study
- Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
- SMN Copy Number Distribution in Mali, West Africa
- STENTRODE™ WITH THOUGHT CONTROLLED DIGITAL SWITCH: An Early Feasibility Study (EFS) of the Safety of an Endovascular Motor Neuroprosthesis (MNP) Device in Participants With Severe Upper Limb Impairment.
- Study of Feasibility to Reliably Measure Functional Abilities’ Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
- Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
- Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
- Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)
- Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
- Sun May Arise on SMA : Newborn Screening of SMA in Belgium
- Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)
- Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
- Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
- Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
- Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
- WREX Outcome Study