National Hospital for Neurology and Neurosurgery,

London WC1N3BG

1 studies enrolling now · 19 studies all time

What they study most

Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Carnitine Palmitoyltransferase (CPT II) Deficiency, Carnitine-acylcarnitine Translocase (CACT) Deficiency, Cerebral Adrenoleukodystrophy (cALD), Fabry Disease, Facioscapulohumeral Muscular Dystrophy (FSHD), Huntington Disease, Huntington's Disease, Huntingtons Disease, Influenza, Late-onset Pompe Disease, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency

Studies at this site

• A Global Prospective Observational Registry of Patients With Pompe Disease — Recruiting now
• A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy — Running, not enrolling
• Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers — Completed
• A Study to Evaluate the Efficacy, Safety and Pharmacokinetics (PK) of a Higher Dose of Ocrelizumab in Adults With Primary Progressive Multiple Sclerosis (PPMS) — Running, not enrolling
• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Running, not enrolling
• A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy — Running, not enrolling
• Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease — Completed
• A Study to Evaluate the Safety and Efficacy of Satralizumab in Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD) — Completed
• Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease — Completed
• A Safety, Reactogenicity, and Immunogenicity Study of mRNA-1045 (Influenza and Respiratory Syncytial Virus [RSV]) or mRNA-1230 (Influenza, RSV, and Severe Acute Respiratory Syndrome Coronavirus 2 [SARS-CoV-2]) Vaccine in Adults 50 to 75 Years Old — Completed
• A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) — Completed
• A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease — Completed
• Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies — Completed
• UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder — Stopped early
• Efficacy and Safety Study of Ozanimod in Relapsing Multiple Sclerosis — Completed
• A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) — Completed
• VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease — Stopped early
• BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) — Stopped early
• A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease — Completed