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Limb-girdle muscular dystrophy (LGMD) is described in the packet definition as a heterogeneous group of muscular dystrophies characterized by proximal weakness affecting the pelvic and shoulder girdles. Cardiac and respiratory impairment may be observed in certain forms of LGMD, according to the same definition. The packet's prevalence field classifies the condition as UNCOMMON, with an estimate of approximately 1 to 9 per 100,000. Disease subtypes listed in the packet include autosomal recessive limb-girdle muscular dystrophy, autosomal dominant limb-girdle muscular dystrophy, and a subtype designated R24.
The packet definition identifies proximal weakness of the pelvic and shoulder girdles as the characteristic feature, with cardiac and respiratory impairment observed in certain forms. No certified phenotype list or symptom frequencies are in the packet.
The packet describes LGMD as a heterogeneous group of disorders. No genes are listed in the packet's known gene field, and no inheritance pattern is certified for the umbrella condition, although subtype names in the packet refer to autosomal recessive and autosomal dominant forms. The genetic basis of individual forms is not described in this packet.
The packet does not certify diagnostic criteria, testing methods, or newborn screening information for limb-girdle muscular dystrophy, so none are described here.
The packet does not certify foundational therapies or FDA-approved treatments for this condition; treatment coverage is not certified in the packet. Orphan drug designations are listed for several products, including Angiotensin (1-7), nandrolone, and prednisone. Each carries status DESIGNATED, which denotes an orphan designation and does not constitute FDA approval.
17 trials found
Natural history and outcome information is not certified in the packet for limb-girdle muscular dystrophy, so no description of course or outcome is given here.
The packet lists clinical trial records for limb-girdle muscular dystrophy, and the search link points to ClinicalTrials.gov. Examples with registry details: NCT05775848, a Phase 3 study of BBP-418 (ribitol) in patients with LGMD2I, sponsored by ML Bio Solutions, Inc., active but not recruiting, 2023-05-31 to 2027-09-01. NCT06246513, a Phase 3 trial of an experimental gene therapy, bidridistrogene xeboparvovec (SRP-9003), for LGMD 2E/R4, sponsored by Sarepta Therapeutics, Inc., active but not recruiting, 2024-01-15 to 2029-11-30. NCT07711730, a telecare psychosocial and cognitive intervention for children and adolescents with LGMD, sponsored by the University of Deusto, recruiting, 2026-03-01 to 2027-06-01.
Data assembled from 5 of 12 sources · Last updated Nov 3, 2026, 8:26 AM UTC
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AI-curated news mentioning limb-girdle muscular dystrophy
Updated Oct 1, 2026
The Speak Foundation has secured a $300,000 commitment from BridgeBio Pharma to enhance its LGMD Centers of Excellence program. This initiative aims to improve specialized care and research for patients with limb-girdle muscular dystrophy, fostering collaboration among patients, clinicians, and industry stakeholders.
The Speak Foundation mobilized LGMD advocates from over 20 states to Capitol Hill, emphasizing the need for policy to align with advancements in rare disease research. This initiative highlights the importance of bipartisan support for limb-girdle muscular dystrophy and other neuromuscular diseases.
The FDA has accepted BridgeBio's application for BBP-418, potentially the first drug for limb-girdle muscular dystrophy, marking a significant advancement in the muscular dystrophy treatment landscape. This priority review reflects the growing momentum in developing therapies for this rare disease.
A national cohort study highlights the association between limb girdle muscular dystrophy and TRIM32 gene variants. This research contributes to the understanding of genetic factors in limb girdle muscular dystrophy.
BridgeBio Pharma's Phase 3 FORTIFY study demonstrates that BBP-418 significantly elevates a key biomarker in patients with limb-girdle muscular dystrophy, supporting a potential launch in early 2027. This data could enhance the drug's profile as it moves closer to market.