Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

JCR Pharmaceuticals Co., Ltd. ( ($JP:4552) ) has provided an update. JCR Pharmaceuticals and Italy’s Italfarmaco have signed an exclusive ...

U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...

TOKYO--(BUSINESS WIRE)--Duchenne Muscular Dystrophy (DMD) patients showed signs of disease progress slowing down, after oral consumption of Neu REFIX ß-glucan for 45 days along with routine ...

Capricor Therapeutics’ Duchenne muscular dystrophy therapy (DMD) appears to have slowed disease progression in older boys and young men who are in the later stages of their disease and unable to walk, ...

• With modern therapeutic strategies to prolong life expectancy, an increasing number of Duchenne Muscular Dystrophy (DMD) patients survive to develop cardiomyopathy and heart failure, resulting in a ...

Investigators evaluated longitudinal MRI and spectroscopy outcomes and ambulatory function among 180 patients with Duchenne muscular dystrophy (DMD) to establish the utility and reproducibility of ...

This announcement follows a bipartisan letter that Senator Collins and a group of nine members of Congress sent last month to ...

CAMBRIDGE, England--(BUSINESS WIRE)--bit.bio, the company coding human cells for novel cures, today launches its first set of muscle cell disease model products designed to advance the discovery and ...

The addition of these two rare diseases to the RUSP is a victory for American families and the nearly 1 in 500 newborns who ...

NASHVILLE, Tenn., Feb. 4, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...