Who is eligible?
We are looking for patients who meet the criteria listed below. If you are not sure whether you are eligible, ask your health care provider or contact the Jain Foundation.
All ambulant and non-ambulant patients.
All patients age 10 and older.
Patients with a confirmed diagnosis of dysferlinopathy, proven EITHER by the identification of two pathogenic dysferlin mutations OR by the identification of one pathogenic dysferlin mutation together with the determination of dysferlin protein expression levels in the disease range as determined by muscle immunoblot or blood monocyte analysis.
- Ability to travel to the nearest study center for the necessary study visits. Patients do not need to live in a study site city in order to participate. Funds are available for patients and a helper if necessary to travel to the nearest study site.