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Top CureDuchenne News

Why Is Capricor Therapeutics Stock Trading Lower On Friday? - Capricor Therapeutics (NASDAQ:CAPR)
FDA · United States

Why Is Capricor Therapeutics Stock Trading Lower On Friday? - Capricor Therapeutics (NASDAQ:CAPR)

67% Center coverage: 6 sources
Capricor Therapeutics Inc. (NASDAQ:CAPR) stock is trading lower on Friday. The company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for cardiomyopathy associated with Duchenne muscular dystrophy (DMD). In the CRL, the FDA stated that it had completed its application review but could not approve…See the Story
Sarepta : Restructuring and Elenidys - Strategic Plan
United States · United States

Sarepta : Restructuring and Elenidys - Strategic Plan

Sarepta: Restructuring and Elenidys – Strategic Plan Sarepta Therapeutics: Restructuring Massive after Deborations in Genic Therapy – Urgent News The pharmaceutical company Sarepta... The post Sarepta: Restructuring and Elenidys – Strategic Plan appeared first on World News.See the Story
Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy – Patient Worthy

Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy – Patient Worthy

Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein. While current standards of care, such as corticosteroids and supportive therapies, have extended patient lifespans and improved quality of life, they remain largely palliative and do not address the root […]See the Story

Kymera Therapeutics Announces Pricing of $250 Million Public Offering

WATERTOWN, Mass., June 26, 2025 (GLOBE NEWSWIRE) — Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of oral small molecule degrader medicines for immunological diseases, today announced the pricing of its underwritten public offering of $250.8 million of shares of its common stock and, in lieu of common stock to […] The post Kymera Therapeutics Announces Pricing of $250 Million Public Off…United States · United StatesSee the Story

Capricor says FDA may not convene expert panel for cell therapy

100% Left coverage: 1 sources
Capricor Therapeutics said on Tuesday the U.S. Food and Drug Administration may not seek its outside experts' advice when deciding on the company's cell therapy for a heart condition associated with Duchenne muscular dystrophy.United States · United StatesSee the Story

The Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy

A panelist discusses how gene therapy using microdystrophin delivered through AAV vectors shows promise for DMD treatment, though challenges remain with delivery efficiency and determining which patients will benefit most.See the Story

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

60% Center coverage: 5 sources
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun 15, 2025-Artificial IntelligenceSee the Story

FDA Meeting Gives Window Into Gene Therapy Field’s Angst

100% Center coverage: 2 sources
Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.FDA · United StatesSee the Story

REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202

100% Left coverage: 1 sources
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for DuchenneAll dose level 2 participants exceeded external natural history controls on all functional measuresBiomarker data demonstrate consistent, robust microdystrophin expression and transduction levels across all treated agesOne new participant aged…See the Story

PepGen to discontinue development of experimental Duchenne therapy; shares drop

100% Center coverage: 1 sources
(Reuters) -Drug developer PepGen said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a mid-stage study. Shares of the company were down 9.5% at $1.43 in extended trading. The therapy PGN-EDO51 did not achieve target levels of dystrophin production. Dystrophin is a protein whose absence leads to muscle weakening in patients with DMD…See the Story

Satellos’ DMD treatment shows efficacy signals in adult patients

Satellos said that following the Phase I data, it will be advancing its DMD small molecule to a Phase II trial.The post Satellos’ DMD treatment shows efficacy signals in adult patients appeared first on Clinical Trials Arena.Irvine, California · IrvineSee the Story

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