MicroRNA-33a and microRNA-33b are encoded within the mammalian SREBF1 and SRBF2 genes, respectively, and both SREBF1 and ...

In total, 11 abstracts on givinostat were accepted for presentation at the Congress, including two for an oral presentation ...

Keywords: Duchenne muscular dystrophy, Becker muscular dystrophy, serum titin, serum creatine kinase, biomarker Citation: Nambu Y, Osawa K, Shirakawa T, Sunami A, Sonehara S, Bo R, Nozu K, Matsuo M ...

For Becker muscular dystrophy, the company reported positive data from MESA, which is an open-label extension trial that provides continued access to sevasemten for participants previously ...

Researchers identified a unique protein signature in the bloodstreams of adults with Becker muscular dystrophy (BMD) and limb-girdle muscular dystrophy (LGMD) that significantly differs from healthy ...

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for sevasemten in Becker muscular dystrophy.

BridgeBio Pharma will host a webinar on limb-girdle muscular dystrophy with Dr. Matthew Wicklund on July 11, 2025.

Edgewise Therapeutics Reports Positive Results on Sevasemten Program for Becker and Duchenne Muscular Dystrophies Jun. 26, 2025 6:00 AM ET Edgewise Therapeutics, Inc. (EWTX) ...

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne.

References [1] Bello L, et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439 [2] van de Velde NM ...