limb-girdle muscular dystrophy type 2L

(redirected from LGMD2L)

limb-girdle muscular dystrophy type 2L

A rare autosomal recessive degenerative myopathy (OMIM:611307) characterised by proximal weakness, weakness of the hip and shoulder girdles, and prominent asymmetrical quadriceps femoris and biceps brachii atrophy.

Molecular pathology
Defects of ANO5, which encodes a calcium-activated chloride channel, cause limb-girdle muscular dystrophy type 2L.
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In addition to the clinical research underway on MYO-101 (see announcement), and this designation for MYO-102, the other candidates include MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. For more information on the MYO-101 trial, visit www.clinicaltrials.gov (study identifier NCT03492346).
Additionally, Myonexus is advancing MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. Like MYO-101, all programs rely upon transfecting a restorative gene utilizing the AAVrh.74 vector.
The company's pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2L).
Myonexus' pipeline also includes MYO-102, a gene therapy candidate for LGMD2D (alpha-sarcoglycanopathy) currently completing a Phase 1/2a clinical trial, MYO-201, a gene therapy candidate for LGMD2B (dysferlinopathy) currently in Phase 1, MYO-103, a preclinical gene therapy candidate for LGMD2C (gamma-sarcoglycanopathy), and MYO-301, a preclinical gene therapy candidate for LGMD2L (anoctamin 5).