Study of ASA-001 for ADSS1 deficiency muscle disease
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This trial tests a drug called ASA-001 (adenylosuccinic acid) for people with a rare genetic muscle disease caused by ADSS1 deficiency. The goal is to see if it is safe and helps muscle function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years or older
- You must weigh between 132 and 187 pounds (60-85 kg)
- You must have a confirmed diagnosis of ADSS1 deficient myopathy with two specific gene mutations
- You must be willing to use effective birth control
- You cannot be pregnant, breastfeeding, or planning to become pregnant
- You must be generally healthy without other serious illnesses or infections
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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