Thelansis

  Limb-girdle muscular dystrophies (LGMDs) are a group of muscle diseases whose mode of inheritance may be either autosomal dominant (LGMD type 1) or autosomal recessive (LGMD type 2). The fukutin-related protein gene (FKRP; OMIM 606596) is implicated in causing both LGMD2I and congenital muscular dystrophy 1C. The FKRP transcript is expressed predominantly in the skeletal muscle, placenta, and heart. Abnormalities in the expression pattern of the α-dystroglycan protein have been demonstrated in muscle biopsies of LGMD2I patients, indirectly suggesting that FKRP, a putative glycosyltransferase, is required for the post-translational modification of dystroglycan. The competitive landscape of Limb-girdle Muscular Dystrophy Type 2i (LGMD2i) includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary. KOLs insights of Limb-girdle Muscular Dystrophy Type 2i ...