Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Equities researchers at Zacks Research lifted their Q4 2024 earnings estimates for Sarepta Therapeutics in a research note issued to investors on Monday, February 3rd. Zacks Research analyst S.

Personalized organoids — ‘mini-organs’ — may accelerate treatment for patients with rare genetic disorders. Courtesy of Europeana via Unsplash Across the globe, countless babies ...

Cumberland Pharmaceuticals今天宣布，其2期临床试验FIGHT DMD获得积极顶线结果。该研究评估了在研创新口服疗法ifetroban治疗杜氏肌营养不良症（DMD）相关心脏疾病的疗效和安全性。心脏疾病是DMD患者的主要死因。新闻稿指出，这项试验是首个专门针对DMD心脏并发症的成功2期临床试验，代表着这一领域的重要突破。

BOSTON - Entrada Therapeutics , Inc. (NASDAQ:TRDA) received authorization from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial for its ...

The Elevate-44-201 study is a global, two-part, randomized, double-blind placebo-controlled phase 1/2 study evaluating the ...

Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...