European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term ...

NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain, July 25, 2025--Prilenia Therapeutics B.V. and Ferrer today ...

CARDIFF, UK / ACCESS Newswire / July 28, 2025 / Familial Adenomatous Polyposis (FAP) is an inherited condition that puts people at a much greater risk of developing colon cancer. If left untreated, ...

Dr. Shaltiel will present the Company’s exosome-based regenerative therapy platform at the Falling Walls Science Summit, taking place in Berlin, Germany from November 6-9, 2025. The winner, selected ...

Two months after laying off 20% of its staff, Korro Bio says it has not retreated from its “3-2-1” corporate strategy as it prepares to release interim clinical data on its lead candidate KRRO-110 in ...

The FDA granted an orphan drug designation to novel hedgehog pathway inhibitor taladegib, also known as ENV-101, for treating ...

BGENE-DMD is designed to treat Duchenne muscular dystrophy (DMD) via a one-time administration in a single AAV.

David Koos, the Chairman and CEO of Regen Biopharma, Inc., discussed the Company’s intention to seek Orphan Drug Designation for HemaXellerate, the Company’s proprietary aplastic anemia cell therapy ...

The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases ...

The pharmaceutical industry has a fresh sense of direction after President Donald Trump’s tax-and-spending law included a ...