Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...

Capricor Therapeutics updates on Deramiocel BLA for duchenne muscular dystrophy: San Diego Thursday, June 26, 2025, 15:30 Hrs [IST] Capricor Therapeutics, a biotechnology company ...

Sarepta Therapeutics (NasdaqGS:SRPT) recently provided updates that may have influenced its stock performance. The announcement of a safety update for ELEVIDYS, concerning severe side effects and a ...

Investing.com -- Edgewise Therapeutics Inc (NASDAQ: EWTX) stock tumbled 7.2% after the company revealed that the U.S. Food and Drug Administration (FDA) deemed its CANYON trial data insufficient for ...

Fintel reports that on June 26, 2025, B. Riley Securities initiated coverage of Capricor Therapeutics (NasdaqCM:CAPR) with a ...

BioMarin’s first quarter performance was shaped by robust global demand for its rare disease therapies, particularly VOXZOGO, ...

Writing in JAMA, four former government officials warn that the Trump administration’s involvement in delaying the approval ...

Edgewise Therapeutics Inc. (NASDAQ:EWTX) saw its shares slide by 7.2% after the U.S. Food and Drug Administration (FDA) ...

SynaptixBio, the only company licensed to commercialise a treatment for a rare but deadly disease, welcomes the announcement but says it lacks a detailed plan The government has announced that every ...

The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.