Studies Open for Recruitment
Duchenne Muscular Dystrophy
Recent Research studies
Study on psychometric properties of "Performance of Upper Limb Module for Duchenne Muscular Dystrophy"(PUL for DMD) in local Chinese population - A Pilot Study -- Ongoing
Clinical trial: Recruitment in Progress
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Viltolarsen(Racer53) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension.
Clinical study: We are recruiting patients to join a , phase 3 clinical trial, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Viltolarsen(Racer53) in Patients.
Criteria:
- 4 to <8 years of age
- Diagnosed with Duchenne mutation that qualifies for exon 53 skipping
- Able to walk and stand without help
Study site: Hong Kong Children’s Hospital
Principal Investigator: Dr. Chan Hoi Shan Sophelia
Clinical Assistant Professor, Department of Paediatrics and Adolescent Medicine, LKS Faculty of Medicine, The University of Hong Kong (Paediatric neurology)
For more detail, please refer to the following factsheet. If you want to join the study, please contact Ms. Amanda Mok (Phone: 22554538).
Viltolarsen FactsheetSpinal Muscular Atrophy
Clinical trial: Closed Recruitment
The phase 3 clinical trial for patient with infant-onset SMA (SMA type 1) has started recruitment in the Department of Paediatrics and Adolescent Medicine , QMH. For more detail, please refer to the Endear website www.smastudy.com. If you like to know more, please contact us.
Clinical trial: Closed Recruitment
The phase 3 clinical trial for patient with late onset SMA (SMA type 2), has completed recruitment in the Department of Paediatrics and Adolescent Medicine, QMH. The study is on going in our hospital.