Limb-Girdle Muscular Dystrophy
Condition Description
Limb-girdle muscular dystrophy is a group of disorders which affect the voluntary muscles around the hips and shoulders. Males and females are affected in equal numbers. Most forms of limb girdle muscular dystrophy are inherited in an autosomal recessive manner. Several rare forms are inherited in an autosomal dominant pattern. There are at least 20 different types of Limb-girdle muscular dystrophy.
Active Trials
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TRIAL TITLE: A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (LGMD2I).
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.
SPONSOR: ML Bio Solutions, Inc.
INDICATION: Limb-Girdle Muscular Dystrophy 2I
STUDY PROTOCOL: MLB-01-005
PHASE: 3
STATUS: Active
ENROLLING PATIENTS: No