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New Developments in the Clinical Care of Limb-Girdle Muscular Dystrophy

New Developments in the Clinical Care of Limb-Girdle Muscular Dystrophy

This article provides an update on recent developments in the clinical care of individuals with limb-girdle muscular dystrophy (LGMD), a group of neuromuscular disorders. It includes expert insights from professionals in the field and highlights key findings from recent studies.

Dr. Peter Kang, director of the Paul and Sheila Wellstone Muscular Dystrophy Center, discussed the current knowledge of LGMD diagnosis and potential therapies. He also addressed the challenges faced by patients and the advancements in care. Dr. Douglas Sproule, chief medical officer at ML Bio Solutions, talked about recent advancements in clinical care and molecular testing for LGMD. He emphasized the importance of gene therapies and the role of industry partners in advancing research.

Dr. Nicholas E. Johnson, division chief of neuromuscular disorders, discussed the advances and challenges in LGMD care and the importance of improving precision genetic diagnoses.

The article also highlights two recent clinical trials. The phase 3 FORTIFY trial, conducted by BridgeBio Pharma, is investigating the efficacy and safety of BBP-418, an oral substrate supplementation therapy for LGMD type 2I/R9. The trial has shown promising results so far, suggesting potential for accelerated approval.

Another trial, the phase 1/2 LION-CS101 trial, is assessing AskBio’s AB-1003 gene therapy for the treatment of LGMD type 2I/R9. The trial aims to deliver a non-mutated version of the FKRP gene using an engineered viral vector. Safety is the primary focus of this trial.

Additionally, the article discusses the findings from a phase 1/2 study of the gene therapy SRP-9003 for LGMD type 2E. The therapy showed sustained protein expression in muscle tissue and improved functional measures in patients.

These recent developments in the clinical care of LGMD provide hope for improved treatments and management strategies for individuals with this rare disease.

Sources:
– NeurologyLive® team
– Peter Kang, MD, FAAN, FAAP
– Douglas Sproule, MD, MSc
– Nicholas E. Johnson, MD, MSci, FAAN
– BridgeBio Pharma
– AskBio
– Sarepta Therapeutics
– (No URLs provided)