News

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Treatment with Translarna (ataluren) increased levels of the protein dystrophin in young boys with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, new clinical trial data show. The findings were presented in a webcast by Stuart W. Peltz, PhD, the CEO of PTC Therapeutics, which…

Mild to moderate isometric leg exercise — contractions of a particular muscle or group of muscles — is safe and improves muscle strength and functional ability in boys with Duchenne muscular dystrophy (DMD) who are able to walk, a study suggests. The study, “Safety, feasibility, and efficacy of…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

In the first year since Parent Project Muscular Dystrophy (PPMD) and Thread launched a mobile app aimed at making participation easier, engagement in the Duchenne Registry — a newly revamped, 12-year-old network of patient-powered data — increased more rapidly than ever before, the non-profit reported. From November…

Applications are now open for the seventh annual Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program for Duchenne muscular dystrophy. The annual program, facilitated by PTC Therapeutics, was designed to help support initiatives that address unmet needs in the Duchenne community. Goals of STRIVE range from…

Parent Project Muscular Dystrophy (PPMD) announced a $1 million investment in Satellos Bioscience to support the development of a technology that aims to repair and regenerate muscle cells in people with Duchenne muscular dystrophy (DMD). “PPMD has been committed to exploring and supporting every single therapeutic possibility. With…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

CureDuchenne has launched a new initiative to prioritize the needs of the Duchenne muscular dystrophy (DMD) community during the COVID-19 vaccine rollout. The nonprofit, which supports patients with DMD, their caregivers, and loved ones, aims to act as an information conduit, educating the DMD community on COVID-19 vaccination,…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…