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Message to the families about trials and their recruitment
Current Trials
- Skip E (18NM19)
Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Further information:
- Baby Skip Extension (19NM20)
An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Further information:
- IONIS/Shine (15NM30)
An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Further information:
- Jewelfish (18NM48)
An open-label study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy
Further information:
- SMA-REACH
Spinal Muscular Atrophy Research and Clinical Hub UK (SMA REACH UK)
Further information:
- North STAR
The U.K. NorthStar Clinical Network - Natural history and standards of care in Duchenne Muscular Dystrophy
Further information:- CMT natural history
A Natural History Study of CMT1B, CMT2A, CMT4A and CMT4C
Further information:- Rare Disease Study
Neuromuscular Rare Disease Translational Research in patients with Duchenne Muscular Dystrophy
Further information:- KineDMD
KINEDMD: A study of kinematics in Duchenne Muscular Dystrophy
Further information:- PTC Therapeutics STRIDE
Long-Term Observational Study of Translarna (Ataluren) Safety and Effectiveness in Usual Care
Further information:- BIND WP5 part 1 (questionnaire study)
Brain Involvement in Dystrophinopathies
Further information:- AFM Natural History Study
Outcome measures in Duchenne Muscular Dystrophy: A Natural History Study
Further information:- Brain DMD
Restoring Dystrophin Expression In Brain To Improve Behavioural And Emotional Problems In Duchenne Muscular Dystrophy
Further information:
- Closed Trials
SIDEROS 16NM17 : A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids.
Sideros-E 18BB36 : A Phase lii Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ldebenone in Patients with Duchenne Muscular Dystrophy {DMD) who completed the SIDEROS study
BMS Spitfire WN40227 17NM18 : A RANDOMIZED, DOUBLE BLIND, PLACEBO-CONTROLLED, STUDY TO ASSESS THE EFFICACY, SAFETY, AND TOLERABILITY OF RO7239361 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY
AveXis LTFU (STR1VE) 17NM10 : Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
SPR1NT (18NM18) 18NM18 : A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-Symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2.
Italfarmaco 16NM23 : Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.
Baby Skip (17NM07) 17NM07 : An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping.
FOR-DMD 12DN09 : Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen.
Vision-DMD (18BB17) : Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD).
Muscle MRI in CMT study 18NM33 :
EmoDe 17NM38 : A Study of Emotional Function in Duchenne Muscular Dystrophy (EmoDe Study)
