Muscular dystrophy is caused by a lack of certain proteins that are needed for muscle structure and function. Specifically, many of the types of MD are caused by a lack of a protein called dystrophin, ...

Performance listed in the above table is net of annual operating expenses. The gross annual expense ratio for the Retail Shares and Institutional Shares as of December 31, 2023 was 1.20% and 0.88% ...

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full ...

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of ...

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle.

The disease mostly affects boys, at a frequency of 1 in 3,500 live births worldwide. DMD is caused by mutations in the DMD gene on the X chromosome, which encodes the dystrophin protein, a crucial ...

Valencia et al. combine elegant in vitro biochemical experiments with functional assays in cardiomyocytes to determine which properties of the FHOD3 formin are essential for sarcomere assembly. Using ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that enrollment and dosing is complete in ...

In this study, the authors show that a long-non coding RNA lncDACH1 inhibits sodium currents in cardiomyocytes by binding to and altering the localization of dystrophin. The authors use a number of ...