A 5-year Natural History Study in LAMA2-related Muscular Dystrophy and SELENON-related Myopathy.
Recruiting
Observational Study
LAMA2-related Muscular Dystrophy
SELENON-related Myopathy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 1 Day – 100
- Sex
- Any
- Study type
- Observational
- Participants needed
- 40 (estimated)
- Sponsor
- Radboud University Medical Center · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
SELENON-related myopathy (SELENON-RM) and LAMA2-related muscular dystrophy (LAMA2-MD) are congenital neuromuscular disorders presenting with slowly, progressive axial muscle weakness, spinal rigidity, scoliosis and respiratory insufficiency. Currently, no curative treatment options exist, yet promising preclinical trials are ongoing. Clinical trials are expected to start within 5 years. Natural hi…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
* Willing and able to complete (part of) the measurement protocol at the Radboudumc, Nijmegen. If patients do not wish or not able to visit our neuromuscular center, they are offered to participate in our study through home visits.
* Genetic conformation of LAMA2-related muscula…
Contacts