- A Novel Approach to Infantile Spasms
- A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
- A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
- Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy
- Addition of Pyridoxine to Prednisolone in Infantile Spasms
- An Open-Label, Single and Multiple Oral Dose Pharmacokinetic Study of Vigabatrin in Infants With Infantile Spasms
- Epilepsy Phenome/Genome Project
- Evaluation of Neuroinflammation in Children With Infantile Spasms
- Genetic Studies in Patients and Families With Infantile Spasms
- Genetics of Severe Early Onset Epilepsies
- Intravenous Methylprednisolone Versus Oral Prednisolone for Infantile Spasms
- Ketogenic Diet vs ACTH for the Treatment of Children With West Syndrome
- Metabolic Abnormalities in Children With Epilepsy
- Molecular Genetics in Infantile Spasms
- Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
- Open-label Extension to Protocol 1042-0500
- Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)
- Prednisolone in Infantile Spasms- High Dose Versus Usual Dose
- Prednisolone Versus Vigabatrin in the First-line Treatment of Infantile Spasms
- Prevention of West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)
- Sabril Patient Registry
- Short-term Ketogenic Diet as Compared With Conventional Long-term Trial in Refractory Infantile Spasms: A Randomized, Controlled Study
- The Effect of Spa and Massage on Babies on Colic Symptoms
- Treatment of Refractory Infantile Spasms With Fenfluramine
- Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
- Trial of the Modified Atkins Diet in Infantile Spasms Refractory to Hormonal Therapy
- Tricaprilin Infantile Spasms Pilot Study
- Use of the Modified Atkins Diet in Infantile Spasms
- Vigabatrin With High Dose Prednisolone Combination Therapy vs Vigabatrin Alone for Infantile Spasm
Clinical Trials
- A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
- A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
- Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
- 2-year Follow-up After a Single Dose Acellular Pertussis Vaccination
- 3-year Follow-up After a Single Dose Acellular Pertussis Vaccination
- 5-year Follow-up After a Single Dose Acellular Pertussis Vaccination
- A Comparison of the Immunogenicity and Safety of Quinvaxem in Mono-dose Vials and Uniject
- A Multi-center Study to Determine the Prevalence and Influence of Pertussis on COPD Exacerbation in Shenzhen
- A Multi-center Study to Determine the Prevalence and Influence of Pertussis on Subacute Cough in Shenzhen
- A Phase 1/2 Clinical Trial of a GamLPV, a Live Intranasal Bordetella Pertussis Vaccine
- A Phase 3 Study of BIBP Diphtheria, Tetanus and Acellular Pertussis (Three Components) Combined Vaccine, Adsorbed
- A Phase I Clinical Study of a GamLPV, a Live Intranasal Bordetella Pertussis Vaccine
- A Phase IV Study to Assess the Safety of EupentaTM Inj
- A Study Exploring Whooping Cough Protection in Children and Adults
- A Study of DTaP-IPV-Hep B-PRP-T Vaccine Given With Prevenar™ and Rotarix™ in Healthy Latin American Infants
- A Study of DTaP//PRP-T Combined Vaccine (ACTACEL) Versus Local DTaP and Act-HIB Monovalent Vaccine in Healthy Infants
- A Study of the Immunogenicity and Safety of Whole-Cell Pertussis Containing Vaccine Administered Concomitantly With RotaTeq™ (V260) or Rotarix™ (V260-036)(WITHDRAWN)
- A Study to Assess the Immunogenicity and Safety of GSK Biologicals’ Infanrix-IPV/Hib Vaccine Administered as a Three-dose Vaccination Course at 3, 4.5 and 6 Months of Age and a Booster Dose at 18 Months of Age in Healthy Infants in Russia
- A Study to Assess the Safety of Adacel® Vaccine
- A Study to Compare LBVD to Eupenta and Imovax Polio in Healthy Adults
- A Study to Determine the Seroprevalence of Bordetella Pertussis in Adults in Hungary
- A Study to Evaluate Safety and Immune Response of Novartis Meningococcal ACWY Conjugate Vaccine In Adolescents
- A Study to Evaluate the Safety and Efficacy(Immunogenicity) of GC3111 in Healthy Adults
- A Trial to Evaluate the Safety and Immunogenicity of ADACEL® Vaccine in Persons 65 Years of Age and Older
- Adacel® Booster Vaccination for CMI Assay Development
- An Immunogenicity and Safety Study of Tetanus, Diphtheria and Acellular Pertussis Vaccine Booster
- Antibody Persistence at Age 3.5 and 4.5 Years After Primary and Booster DTaP-IPV-Hep B-PRP~T or Infanrix Hexa Vaccination
- Antibody Persistence in Healthy Children After Primary and Booster DTaP-IPV-Hep B-PRP-T Vaccine or Control Vaccine
- Assess Immune Response Following Primary Vaccination With Tritanrix™-HepB Vaccine Mixed With 3 Formulations of Hib-MenAC Vaccine to Infants
- Assessing Contacts’ Decision Making on Reducing the Risk of Pertussis Transmission to Newborns Through Immunisation
- Assessment of the Immunogenicity and Safety of PENTAXIM™ in Philippines
- Assessment of the Reactogenicity of ADACEL® (TdcP Vaccine) in Children and Adolescents 7 to 19 Years of Age
- Assessment, Feedback, Incentive, Exchange (AFIX OB) A Customizable Quality Improvement Intervention to Increase Maternal Vaccine Uptake
- Booster Study of Combined Diphtheria-tetanus-acellular Pertussis Vaccine in Healthy Adults
- Boostrix® Pregnancy Registry
- Bordetella Pertussis Colonisation Challenge Study
- CD8 Reactivity to Microorganisms in Blood and Breast Milk
- Comparison of a DTaP-IPV-HB-PRP~T Combined Vaccine to Infanrix™-Hexa, When Administered With Prevnar® in Thai Infants
- Comparison of DTaP-HB-PRP~T Combined Vaccine to Tritanrix-HepB/Hib™, Both Given Concomitantly With Oral Polio Vaccine
- Comparison of DTaP-IPV-Hep B-PRP~T Combined Vaccine to CombAct-HIB® Concomitantly Given With Engerix B® Paediatric and OPV
- Comparison of Safety and Immunogenicity of Pediacel™ and Infanrix™ IPV+Hib (Penta) Given in a 3 Dose Schedule in Infants
- Concomitant Use of Gardasil (V501, Human Papillomavirus [Types 6, 11, 16, 18] Recombinant Vaccine) With Combined Diptheria, Tetanus, Pertussis and Poliomyelitis Vaccine in Adolescents (V501-024)(COMPLETED)
- Concomitant Use of Gardasil (V501) (Human Papillomavirus [Types 6, 11, 16, 18] Recombinant Vaccine) With Combined Diphtheria, Tetanus, Pertussis Vaccine and Meningococcal Conjugate Vaccine in Adolescents (V501-025)
- Confirmatory Study of BK1310 in Healthy Infants
- Database Surveillance Safety Study of PENTACEL® Vaccine
- Descriptive, Open-label, Multicenter Study of the Safety of Redosing With ADACEL® Vaccine
- Descriptive, Post-marketing, Passive Surveillance Safety Study of ADACEL™ Vaccine
- DTaP-IPV-HB-PRP-T Combined Vaccine as a Primary Series and a Second Year of Life Booster in HIV-Exposed Infected and Uninfected Infants
- DTaP-IPV-Hep B-PRP~T Combined Vaccine Versus DTaP-IPV//PRP~T Combined Vaccine + Hep B Vaccine in Hep B Primed Infants
- DTaP-IPV/Hib Vaccine Primary & Booster Vaccinations Versus Co-administration of DTaP-IPV and Hib Vaccine in Japanese Infants
- Effectiveness of a Vaccination Program in the Community Ob/Gyn Setting
- Effectiveness of Maternal Immunization With Boostrix at Preventing Pertussis Among Infants
- Establishing a Controlled Human Infection Model of Bordetella Pertussis
- Evaluating a Caregiver SMS Reminder Intervention to Reduce Immunization Drop-out in Arua, Uganda
- Evaluation of an Infant Immunization Encouragement Program in Nigeria
- First Adult Safety Trial on Nasal Live Attenuated B. Pertussis Vaccine
- Gambia Pertussis Study (GaPs)
- How Does the Clinical Tool ‘What’s Going Around’ Affect Clinical Practice
- Immune Memory Foll Pry Vaccination With DTPw-HBV/Hib Vaccine Formulation; Immuno & Reacto of Booster Dose at 15-18 Mths
- Immune Responses in Adults to Revaccination With ADACEL® 10 Years After a Previous Dose
- Immunising Mums Against Pertussis 3
- Immunogenicity and Reactogenicity of DTPa-HBV-IPV/Hib, Compared to DTPa-HBV-IPV and Hib Administered Separately
- Immunogenicity and Safety of Adacel Polio Vaccine
- Immunogenicity and Safety of ADACEL™ as Fifth Dose in Taiwan
- Immunogenicity and Safety of DTPa-HBV-IPV/Hib Compared to DTPa-IPV/Hib and HBV Administered Concomitantly
- Immunogenicity and Safety of Pentaxim as 3 Doses Primary Vaccination Followed by a Booster Dose at 18 Months
- Immunogenicity and Safety of Pentaxim in South African Infants
- Immunogenicity and Safety of Pentaxim™ in an Indian Population
- Immunogenicity and Safety of Sanofi Pasteur Pentaxim Combined Vaccine in Infants in Thailand
- Immunogenicity and Safety of Sanofi Pasteur’s Combined Vaccine Given as a Three-Dose Primary Series at 2, 3,4 Months of Age and Followed by a Booster Dose Given at 16 to 17 Months of Age in Vietnamese Infants Who Previously Received a Dose of Hepatitis B Vaccine at Birth or Within 1 Week After Birth
- Immunogenicity and Safety of Tetraxim Versus Local DTP + IPV
- Immunogenicity and Safety of TETRAXIM™ Given as a Booster Dose at 4 to 6 Years of Age
- Immunogenicity and Safety Study of a Booster Dose (5th) of Diphteria-Tetanus-Pertussis-Polio Vaccine
- Immunogenicity and Safety Study of Proquad® and Infanrix® Hexa When Administered Concomitantly (V221-035)
- Immunogenicity of Adacel® and BOOSTRIX® Vaccines in Adolescents
- Immunogenicity of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared With PENTAXIM™ and ENGERIX B® at 2-3-4 Months Schedule
- Immunogenicity Study of Antibody Persistence and Booster Effect of DTaP-HB PRP~T Combined Vaccine in Filipino Infants
- Immunogenicity Study of Antibody Persistence and Booster Effect of DTaP-HB-PRP~T Combined Vaccine or Tritanrix-HepB/Hib™
- Immunogenicity Study of Antibody Persistence and Booster Effect of PENTAXIM™ at 18 Months in Healthy Argentinean Infants
- Immunogenicity, Safety and Tolerability of a Fully Liquid Pentavalent (DTwP-Hib-Hep B) Vaccine in Infants 6-8 Weeks of Age
- Immunology of Non-specific Effects of Vaccine
- Increasing Vaccine Uptake Among Veterans at the Atlanta VA Health Care System
- Influence of BCG on TDaP-IPV Vaccination
- Intravenous Pertussis Immune Globulin in Patients With Severe Childhood Pertussis Infection
- Long-term Persistence of Hepatitis B and Pertussis Antibody Responses in Healthy 4 to 5 Year Old Children Previously Vaccinated With Vaxelis® or INFANRIX® Hexa (V419-012)
- Lot Consistency Study of DTaP-IPV-HB-PRP~T Vaccine Administered at 2-4-6 Months of Age in Healthy Infants
- Maternal Antibody in Milk After Vaccination
- Maternal Pertussis Wholecell Responses
- Maternal Tdap Immunization in Guatemala
- Mobile Phone SMS Messages and Automated Calls in Improving Vaccine Coverage Among Children in Pakistan
- Montelukast for Persistent Cough in Young People and Adults
- Observational Study Describing the Immune Profile Induced By Pertussis Vaccines
- Optimising the Timing of Whooping Cough Immunisation in MUMs
- Pathogenic Bordetella Rapid Detection
- Pentavalent DTaP-Hep B-IPV
- Persistency Study After aP / Tdap Booster Vaccines in Adult Subjects (V113_01 Extension 1)
- Pertussis (Tdap) Vaccination in Pregnancy
- Pertussis Acellular Vaccine Adjuvanted With TQL1055
- Pertussis and Meningitis C Concomitant Vaccination in Adolescents
- Pertussis Immunization During Pregnancy & HIV Infection
- Pertussis Immunization During Pregnancy: Effect in Term and Preterm Infants
- Pertussis Infection in Adolescents and Adults With Prolonged Cough
- Pertussis Vaccination in Pregnant Women
- Pertussis Vaccine in Healthy Pregnant Women
- Phase I Clinical Trial in Healthy Children
- Phase III Randomized, Double-Blind, Placebo-Controlled Study of Acellular and Whole-Cell Pertussis Vaccines
- Phase III Randomized, Double-Blind, Placebo-Controlled Study of Acellular and Whole-Cell Pertussis Vaccines
- Phase IV Interchangeability Study of a Liquid Pentavalent Combination Vaccine
- Pilot Study Freeze and Transport Immune Cells
- Pneumococcal Vaccine and Routine Pediatric Immunizations in HIV-Infected Children Receiving Anti-HIV Drugs
- Post Marketing Surveillance for ADACEL™ in South Korea
- Post-authorization Safety Study of Euforvac-Hib Vaccine for Active Primary Immunization in Infants From 6 Weeks
- Post-licensure, Modified Double-blind, Multi-center Study Evaluating Safety and Immunogenicity of ADACEL® and BOOSTRIX®
- Post-marketing Evaluation of Reactions Following Receipt of Recommended Adolescent Pertussis Vaccine
- Protecting Pregnant Women From Infectious Diseases
- Randomized Controlled Trial of the Impact of Mobile Phone Delivered Reminders and Travel Subsidies to Improve Childhood Immunization Coverage Rates and Timeliness in Western Kenya
- Rates of Pertussis Disease Among Persons Receiving Pentacel® or Other Pertussis Vaccines
- Regulatory Post-Marketing Surveillance Study for TETRAXIM™
- Retrospective Survey of Safety of Fourth Dose Pentacel® in Children
- Safety and Dose Ranging Study of Acellular Pertussis and Acellular Pertussis -Tetanus-Diphtheria Booster Vaccines in Healthy Adults Ages 18 to 40 Years
- Safety and Immune Response of Different Pediatric Combination Vaccines.
- Safety and Immunogenicity in Adults of Revaccination With Adacel® Vaccine 10 Years After a Previous Dose
- Safety and Immunogenicity of 2 Doses Versus 1 Dose of Acellular Pertussis Vaccines Containing Genetically-detoxified Pertussis Toxin in Young Adults Previously Primed With Acellular Pertussis Vaccines
- Safety and Immunogenicity of DAPTACEL® as 5th Dose in Children 4 to 6 Years Old After 4 Doses of Pentacel™ or DAPTACEL®
- Safety and Immunogenicity of Hexavalent Vaccine(DTwP-HepB-IPV-Hib) in Healthy Infants
- Safety and Immunogenicity of Intranasal BPZE1 Vaccination in Healthy Adults
- Safety and Immunogenicity of Tdap Vaccine Compared to DTaP Vaccine in Children 4 to 6 Years of Age
- Safety and Immunogenicity Study of a Diphtheria, Tetanus, Acellular Pertussis and Poliomyelitis Vaccine (REPEVAX) Followed by 2 Doses of a Diphtheria, Tetanus and Poliomyelitis Vaccine (REVAXIS) in Subjects of 40 Years of Age or Older
- Safety and Immunogenicity Study of a Liquid Pentavalent Combination Vaccine
- Safety and Immunogenicity Study of Concomitant Versus Non Concomitant Administration of a Diphtheria, Tetanus, Pertussis and Poliomyelitis Vaccine (REPEVAX) and Influenza Vaccine (VAXIGRIP) in Subjects Aged From 60 Years Old
- Safety and Immunogenicity Study of Tetanus, Diphtheria and Acellular Pertussis (Tdap) Vaccine
- Safety of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared to Tritanrix-HepB/Hib™ and OPV Given at Age 2, 4, and 6 Months.
- Safety of PENTAXIM® Given as a Three-Dose Primary Vaccination at 2, 3, and 4 Months of Age in Infants in China
- Safety, Long Term Immunogenicity and Lot Consistency Study of Liquid Pentavalent Combination Vaccine
- Sanofi Pasteur’s Tdap Combined Vaccine as a Booster Versus Local DT Vaccine in Children or Versus Local Td Vaccine in Adolescents and Adults in China.
- Sero-prevalence of Anti-pertussis Antibodies and Disease Awareness Among Spanish Healthcare Professionals
- Seroimmunity 2007 and Sub Study of the Swedish Population Regarding Vaccine Preventable Disease
- Seroprevalence of Bordetella Pertussis Antibodies and Anti-pertussis Antibodies Response After a Single Dose of Tdap Vaccine in Thai Pregnant Women
- Seroprevalence of Pertussis Among Healthy Children and Adolescents in Kazakhstan
- Sources of COmplement in Meningococcal and Pertussis Serum Bactericidal Antibody Assays
- Study Comparing a DTaP-HB-PRP~T Combined Vaccine With Tritanrix HepB/Hib™, Concomitantly With OPV in Healthy Infants
- Study of Adacel® Vaccine Administered to Persons 10 Years of Age
- Study of Adsorption Tetanus-diphtheria-acellular Pertussis (DTaP) Vaccine in Healthy 3 to 5 Months Infants
- Study of an Investigational Vaccine in Healthy Infants in Taiwan (V441-001)(COMPLETED)
- Study of BK1301 (DTaP Vaccine) as a Booster in Adolescents
- Study of BPZE1 (High Dose) Nasal Live Attenuated B. Pertussis Vaccine
- Study of BPZE1 Intranasal Pertussis Vaccine (Administered Via VaxINator(TM)), Prime + Boost, in Healthy Adults
- Study of DAPTACEL® Administered as a 5th Dose in 4 to 6-Year-Old Children Previously Immunized With PENTACEL™
- Study of DTap-IPV Compared to DAPTACEL® and IPOL® as the 5th Dose in Children 4 to 6 Years of Age
- Study of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared to Infanrix®Hexa in Healthy Peruvian Infants
- Study of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared With PENTAXIM™ and ENGERIX B® PEDIATRICO in Argentinean Infants
- Study of DTaP-IPV-Hep B-PRP~T Combined Vaccine in Indian Infants Previously Given a Dose of Hepatitis B Vaccine at Birth
- Study of Immunogenicity and Safety of a Booster Dose of DTaP-IPV-HB-PRP~T Combined Vaccine in Healthy Turkish Infants
- Study of Menactra® in Children Aged 4 to 6 Years When Administered Concomitantly With a Fifth Dose of DAPTACEL®
- Study of Menactra® in Healthy Subjects at 9 Months and Concomitantly With Pentacel® at 15 to 18 Months of Age
- Study of Menactra® in US Adolescents When Administered Concomitantly With Tdap Vaccine
- Study of PENTAXIM™ Vaccine Versus TETRAXIM™ Vaccine Given With ACTHIB™ Vaccine in South Korean Infants.
- Study of PR5I, a Pediatric Combination Vaccine With Enhanced Hepatitis B Component Given Concomitantly With Prevnar®
- Study of Quinvaxem for Vaccination Against Diphtheria, Pertussis, Tetanus, Hepatitis B and Diseases Caused by Haemophilus Influenzae Type B
- Study of SP306 Given Intramuscularly Compared to DT Given Subcutaneously in Japanese Adolescents 11 – 12 Years Old
- Study of Tetanus and Diphtheria Toxoids Adsorbed Combined With Component Pertussis Vaccine and Inactivated Poliomyelitis
- Study of Tetanus Toxoid, Reduced Diphtheria Toxoid and Acellular Pertussis Vaccine (ADACEL®) as a Booster in Adolescents
- Study of Tetanus Toxoid, Reduced Diphtheria Toxoid, and Acellular Pertussis Vaccine Adsorbed in Healthy Subjects
- Study of the Booster Effect of DTaP-IPV-Hep B-PRP~T Combined Vaccine or Infanrix Hexa™ and Prevenar™ in Healthy Infants
- Study of the DTaP-IPV-Hep B-PRP~T Combined Vaccine Following a Primary Series of DTacP IPV-HepB-PRP-T or Infanrix Hexa™
- Study of the Effect of Pneumococcal Conjugate Vaccine (PCV) on Immunogenicity of Pentacel™
- Study of the Environmental Factors Modulating Children Immune Response in Northern Senegal
- Study of the Safety, Immunogenicity and Lot Comparability of DAPTACEL When Administered With Other Recommended Vaccine
- Study of the Tdap Combined Vaccine (ADACEL™) as a Booster Dose in Healthy Adults and Children in China
- Study on the Safety and Immunogenicity of Boostrix Vaccine in Pregnant Malian Women and Their Infants
- Study to Assess if Quinvaxem Can be Interchanged With Other Pentavalent Vaccines During Standard Childhood Vaccination
- Study to Compare Pediacel® to Infanrix®-IPV+Hib When Both Are Co-Administered With Prevenar® in Infants and Toddlers
- Study to Evaluate the Immunogenicity and Safety of LBVD (Hexavalent Vaccine; DTwP-HepB-IPV-Hib Vaccine), Given to Healthy Infants at Primary Series
- Surveillance Program to Determine Product Specific Rates of Invasive Hib Disease
- TDAP Safety in Pregnant Women
- Tdap Vaccination for Infant Caregivers
- Tdap Vaccine in Post-Partum Women
- Testing the Use of Prompts to Increase Adolescent Immunization Rates
- The Feasibility of Pertussis Immunization in a Canadian Emergency Department
- The PertADO Geneva Trial
- The Safety and Immunogenicity of Acellular Pertussis Vaccine in Bone Marrow Transplant Recipients
- The Safety of Boostrix Following Routine Immunization of Pregnant Women
- The Transparent Safety Study of the 5 in 1 (DTwP-rHepB-Hib) Combination Vaccine Produced by the Indian Serine Institute in Children of Viet Nam Healthy From 6 to 12 Weeks of Age in a 3-dose Regimen, the Interval Between Doses is 4 Weeks
- To Evaluate the Immunogenicity and Safety of DTaP-IPV Vaccine Administered as a Boosting Dose to Healthy Children of 4-6 Years
- Vaccine -diphthEria -tetaNus -Acellular pertUssis-inactivated polioviruS
- Vaccine Responses in Infants After Acellular Pertussis Vaccination During Pregnancy in Thailand
- Vanderbilt Pertussis Exposure Study: PEP in Vaccinated Healthcare Workers Following Pertussis Exposure
- Viaskin Pertussis Vaccine Trial
- Vouchers to Promote Tdap Vaccination
- Buspirone Treatment of Anxiety in Williams Syndrome
- Cognitive and Behavioral Therapy of Anxiety in Williams Syndrome
- Defining the Brain Phenotype of Children With Williams Syndrome
- Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
- Fat Distribution and Glucose Metabolism in Williams Syndrome
- Impact of Elastin Mediated Vascular Stiffness on End Organs
- Molecular Characterization of Patients Affected by Williams Syndrome and Autism.
- Motor Deficits and Signal Conduction in Individuals With Williams Syndrome
- Observational Study of Advanced Data Analytics in Genetic Conditions
- Quantification of Elastin Markers Synthesis in Williams-Beuren Syndrome and 7q11.23 Micro-duplication Syndrome
- Response Inhibition Training for Children With Williams Syndrome
- Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes
- Vitamin D Metabolism and the Williams Syndrome
- Williams Syndrome (WS) and Supravalvular Aortic Stenosis (SVAS) DNA and Tissue Bank
- Williams Syndrome Strength, Hormones, Activity & Adiposity, DNA Programming, Eating Study
- WS-SAVE Study (Williams Syndrome Skin and Vessel Elasticity Study)
- A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
- A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson’s Disease Patients
- A Phase I/II Study of VTX-801 in Adult Patients With Wilson’s Disease
- A Registered Cohort Study on Wilson’s Disease
- Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
- Cardiac Involvement in Wilson’s Disease
- Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
- Clinical Features and Outcome of Wilson’s Disease With Generalized Epilepsy in Chinese Patients
- Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease
- Cohort Research on Wilson’s Disease
- Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
- Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
- Early Access Program for ALXN1840 in Patients With Wilson Disease
- Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease With an Extension Period of up to 60 Months
- Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients
- Efficacy and Safety Study of Zinc Acetate to Treat Wilson’s Disease in Japan.
- Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson’s Disease in Japan.
- Efficacy of Invitro Expanded Bone Marrow Derived Allogeneic Mesenchymal Stem Cell Transplantation Via Portal Vein or Hepatic Artery or Peripheral Vein in Patients With Wilson Cirrhosis
- Establishment of Human Cellular Disease Models for Wilson Disease
- Evaluation of Patients With Liver Disease
- ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders
- Gandouling in the Treatment of Wilson’s Disease
- Inhibitory rTMS in Dystonic Wilson Patients
- International Wilson’s Disease Patient Registry (iWilson Registry)
- Natural History of Wilson Disease
- Oral Health and Wilson’s Disease: SOMAWI
- Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants
- Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.
- Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson’s Disease-related Liver Failure
- Real World Evidence Study in Subjects With Wilson’s Disease
- Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease
- rTMS in Wilmson Diasease Dysarthria
- sCD163 and sMR in Wilsons Disease – Associations With Disease Severity and Fibrosis
- Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
- Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants
- Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
- Study of Retinal Vascular Parameters in Patients With Wilson’s Disease
- Study of Tetrathiomolybdate in Patients With Wilson Disease
- Study of Zinc for Wilson Disease
- Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
- The Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment
- The Individual Therapy for Patients With Wilson’s Disease
- Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson’s Disease
- Wilson France Register
- WILSTIM – DBS (WILson STIMulation – Deep Brain Stimulation)
- A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
- A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
- Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
- Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells for Patients With Primary Immunodeficiencies
- Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
- Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
- Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
- Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
- Gene Therapy for WAS
- Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
- Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
- Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
- Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
- Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
- Molecular and Clinical Studies of Primary Immunodeficiency Diseases
- Participation in a Research Registry for Immune Disorders
- Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
- Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
- Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
- Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients
- Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
- Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
- Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
- Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
- Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
- Accessory Pathway Antegrade Effective Refractory Period Among WPW Patients: the Risk in Relation to the Location
- Cryotherapy Versus Radiofrequency Ablation for Treatment of Para-hisian Accessory Pathways
- Risk Assessment in Patients With Symptomatic- and Asymptomatic Preexcitation
- Routine Mini-invasive Electrophysiology Study for Patients Feeling Tachycardia, With a Negative Holter ECG
- Stress ECG Test for the Evaluation of the Risk of Sudden Cardiac Death in a Paediatric Cohort With WPW Pattern
- Wolff-Parkinson-White Syndrome Anterograde Refractory Period of Accessory Duct
- A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
- A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
- Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
- Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
- GLP Analogs for Diabetes in Wolfram Syndrome Patients
- I-Tracking Neurodegeneration in Early Wolfram Syndrome
- Monogenic Diabetes Misdiagnosed as Type 1
- Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1
- Tracking Neurodegeneration in Early Wolfram Syndrome
- Wolfram Syndrome International Registry and Clinical Study
- A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
- Breakfast for Young Women
- Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
- In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
- Lysosomal Acid Lipase (LAL) Deficiency Registry
- Prevalence and Risk Factors of Women Mental Health Disorders
- Registry of Patients Diagnosed With Lysosomal Storage Diseases
- Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
- Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up
- A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
- A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
- A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants
- A Phase III Trial of Lorenzo’s Oil in Adrenomyeloneuropathy
- A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
- A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
- A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
- Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
- Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
- Baby Detect : Genomic Newborn Screening
- Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children
- Early Diagnosis Of Childhood Cerebral ALD
- Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
- Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy
- Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
- Expanded Access for Lorenzo’s Oil (GTO/GTE) in Adrenoleukodystrophy
- Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
- HSCT for High Risk Inherited Inborn Errors
- Human Placental-Derived Stem Cell Transplantation
- Lentiviral Gene Therapy for X-ALD
- Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
- Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
- MD1003-AMN MD1003 in Adrenomyeloneuropathy
- Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank
- Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy and Metachromatic Leukodystrophy
- MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
- Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children
- Newborn Screening for Adrenoleukodystrophy
- Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
- Plasma Exchange With Albumin in AMN Patients
- Precision Exercise in Children With Malignant Hemopathies
- Quality of Life in Women With X-linked Adrenoleukodystrophy
- Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
- Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
- Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
- Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
- SMART-ALD – A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)
- Stem Cell Transplant for Inborn Errors of Metabolism
- Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
- Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
- Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
- The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
- Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
- A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
- Assessment of Immunogenicity of Zostavax® in Patients With Antibody Deficiency 60 Years of Age and Older
- Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases
- COVID-19 Vaccine Responses in PIDD Subjects
- Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
- Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases
- Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
- Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy
- A Cross-Sectional Natural History Study to Evaluate Sweat Volume and Other Phenotypic and Genetic Characteristics in Patients Affected by XLHED
- A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200
- Characterization of Sweat Gland Function in Patients With Recessively Inherited Hypohidrotic Ectodermal Dysplasia
- Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected by Hypohidrotic Ectodermal Dysplasia – A
- Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia (ECP-012)
- Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002
- Impact of Dysregulation of Core Body Temperature on Sleep in Patients With Hypohidrotic Ectodermal Dysplasia
- Intraamniotic Administrations of ER004 in Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia
- Investigation of Chronic Inflammatory Processes in Male Individuals With Hypohidrotic Ectodermal Dysplasia
- Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia: Intrafamilial Variation
- Medical Record Review of Hypohidrotic Ectodermal Dysplasia Clinical Phenotype
- Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia
- Pediatric SARS-CoV-2 Infections: Course of COVID-19, Immune Responses, Complications and Long-term Consequences
- Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
- Phenotypic and Genetic Properties in Males at Risk for X-linked Hypohidrotic Ectodermal Dysplasia: Evaluation of an Early Diagnosis Technology and Tests to Assess Nutritional Status
- Phenotypic Properties in Individuals Affected With XLHED
- Short Term Effects and Risks of Physical Exercise in Subjects With Hypohidrotic Ectodermal Dysplasia
- Sweat Duct Imaging in Mother/Newborn Dyads
- X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) Carrier Outlook Toward Reproduction Survey