Daughter Verena shares her personal story of living with Limb-girdle type 2A muscular dystrophy. The progressive disease affects muscles around the shoulders, limbs, hips, and thighs, making simple ...
– U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5 – Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 – Data expected for SRP-9003 ...
-- Continued functional improvements wereobserved at 18 months in the low-dose cohort-- -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration -- -- ...
It's not easy to distinguish between the dozens of subtypes of limb-girdle muscular dystrophy - a rare, genetic muscle disease characterized by weakness in the hips and shoulders that causes ...
PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced ...
BridgeBio Pharma, Inc. announced it will host an investor webinar on July 11, 2025, at 8:00 am ET, featuring Dr. Matthew Wicklund from the University of Texas Health Science Center. The webinar will ...
The advent of gene therapies, which can target specific variants, means pinpointing the genetic roots of each patient’s disease has taken on new importance, researchers said. A team of researchers ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that screening is underway in Study ...
EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today ...
To continue reading this content, please enable JavaScript in your browser settings and refresh this page. Preview this article 1 min The search for a treatment for ...
Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing ...
Eleven-year-old Charlotte Colella and her mother Alexa Colella pose in front of merchandise available for their fundraiser, set for 6 p.m. Tuesday at downtown Champaign's Fire Doll Studios.
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