Nemaline myopathy study for children and teens
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study creates a network for children with nemaline myopathy (a muscle weakness condition). It aims to better understand the condition and prepare for future treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Aged 0 to 18 years old
- Diagnosed with nemaline myopathy, confirmed by a genetic test for ACTA1 or NEB gene changes
- Patient and parent or guardian agree to join the study
- Not currently in another treatment trial that tests new therapies
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study follows people with nemaline myopathy over time to better understand how the disease progresses. It helps researchers learn more about the condition and plan future treatments.
This study follows people with nemaline myopathy over time to understand how the condition progresses naturally. It does not test a treatment, so it helps researchers learn more about the disease.
This study follows patients with congenital myopathies to understand how their muscles get tired over time. It may help develop better treatments.
This study looks at how children and teens with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk, by having them do walking tests while wearing sensors. The goal is to find better ways to track how these diseases progress and how treatments work over time, without needing blood tests or scans.
This study follows people with a rare genetic muscle condition called TNNT1 myopathy over time. Researchers will track how the disease progresses to better understand it and help plan future treatments.
This study looks at children with inherited muscle diseases (not Duchenne muscular dystrophy) to understand their symptoms and muscle function better. It may help doctors identify what type of muscle disease a child has and how it affects them day-to-day.
Hear when a new Nemaline Myopathy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.