Confounding data from a Pfizer clinical trial has rattled the field of gene therapy for Duchenne muscular dystrophy, raising more questions about the regulatory standard used to approve a ...

The rise comes a day after the company said it was expanding its pipeline to include two new candidates targeting rare ...

An FDA expansion of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) Elevidys has sparked a new surge in sales for the ...

ATA-200 is a one-time gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a serious and debilitating condition that affects children and ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment ...

Analyst Joseph Schwartz of Leerink Partners reiterated a Buy rating on PepGen Inc. (PEPG – Research Report), with a price target of ...

“We need help from other people,” pleaded Pavlina. “We saw videos from the children who were already there, it [the treatment ...

The global Duchenne muscular dystrophy (DMD) therapeutics market is set to grow by $4.4 billion from 2024 to 2028, with a ...

Investors consider Dyne Therapeutics' treatments for muscle diseases and strong cash position as a speculative Buy ...

(AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease ...

Research Triangle Park, North Carolina Saturday, November 9, 2024, 13:00 Hrs [IST] ...