Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...

Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

Analyst Michael Ulz of Morgan Stanley maintained a Buy rating on Dyne Therapeutics (DYN – Research Report), retaining the price target of ...

Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 -WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, ...

Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation - CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Solid ...

Stifel analysts began coverage of Edgewise Therapeutics (NASDAQ:EWTX), currently valued at $2.6 billion, with a Hold rating and set a price target of $30.00. According to InvestingPro data, the stock ...

Oppenheimer reiterated its Outperform rating on Dyne Therapeutics (NASDAQ:DYN), maintaining a $60.00 price target, ...

This year, my colleagues have selected me to lead the Senate Armed Services Committee (SASC). I am eager to take on the responsibilities of the role, because the committee’s work ...

Below is a review of novel drug and biological products approved by the Food and Drug Administration (FDA) in 2024. The list includes several groundbreaking therapies that are expected to change the ...

A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...

Dillon, left, as Supergirl, Dominique Dickson as Wonder Mom and Dason as Raphael the Ninja Turtle got into the spirit of ...