Breakthrough data from Vatiquinone trials propels PTC Therapeutics stock to 52-week high

Pallavi Madhiraju- October 8, 2024 0

PTC Therapeutics Inc. (NASDAQ: PTCT) is experiencing significant momentum within the biotechnology sector following a surge in its stock price to a 52-week high of ... Read More

Capricor Therapeutics announces promising long-term results for deramiocel deramiocel in Duchenne muscular dystrophy

Pallavi Madhiraju- June 29, 2024 0

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company at the forefront of developing cell and exosome-based therapies, has shared encouraging three-year safety and efficacy results from ... Read More

FDA approves Italfarmaco’s Duvyzat for duchenne muscular dystrophy treatment

Pallavi Madhiraju- March 24, 2024 0

In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S. Food and Drug Administration (FDA) approval of ... Read More

FDA advances Sarepta Therapeutics’ ELEVIDYS for Duchenne muscular dystrophy treatment

Pallavi Madhiraju- February 17, 2024 0

In a promising development for the treatment of rare diseases, Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a frontrunner in precision genetic medicine, announced that the U.S. Food ... Read More

IPS HEART gets third FDA pediatric rare disease designation for ISX9-CPC

Pallavi Madhiraju- July 9, 2023 0

IPS HEART, a private cell therapy company developing treatments for Duchenne muscular dystrophy and heart failure, announced that it has received a third Rare Pediatric ... Read More

FDA committee backs approval of Sarepta Therapeutics’ SRP-9001 for DMD

Pallavi Madhiraju- May 13, 2023 0

Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) of the US Food and Drug ... Read More

Sarepta Therapeutics to seek FDA accelerated approval for SRP-9001 in DMD

pallavi123- August 1, 2022 0

Sarepta Therapeutics said that it plans to file a biologics license application (BLA) with the US Food and Drug Administration (FDA) to seek accelerated approval ... Read More

ReveraGen, Santhera get FDA grant for BMD trial of vamorolone

pallavi123- September 27, 2021 0

ReveraGen Biopharma and Santhera Pharmaceuticals have secured a $1.2 million grant for funding their clinical trial of vamorolone in Becker muscular dystrophy (BMD) from the ... Read More

PTC Therapeutics wins Russian approval for Translarna for nmDMD

pharmanewsdaily- December 6, 2020 0

US-based PTC Therapeutics has secured marketing approval in Russia for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The approval for ... Read More

Roche signs $2.85bn deal with Sarepta for SRP-9001 DND gene therapy

pharmanewsdaily- December 24, 2019 0

Roche has signed a licensing deal worth up to $2.85 billion with Sarepta Therapeutics for the exclusive ex-US commercial rights to the latter’s SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), ... Read More