This study is NOT currently recruiting. 

Description: This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the significant majority of existing diagnosed patients based upon the established epidemiology of the disease.

Eligibility & Criteria 

IRB #:  IRB00382616
 
Principal Investigator: Dr. Doris Leung
 
Eligible Age Range:  12-60

Gender:  All

Diagnosis:  Limb Girdle Muscular Dystrophy 21 (LGMD21)