News

February 4, 2021February 4, 2021

Isometric Exercise in DMD Found to Improve Boys’ Strength, Function

News

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 ... Read more

February 3, 2021February 3, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

News, syndicated

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 ... Read more

February 2, 2021February 2, 2021

New App Plays ‘Pivotal’ Role in Increasing Registry Successes

News

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 ... Read more

January 28, 2021January 28, 2021

STRIVE Applications for Duchenne Community Now Open

News

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, ... Read more

January 26, 2021January 26, 2021

PPMD Invests $1M in Satellos With Goal of Repairing Muscle Cells in Duchenne

News

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 ... Read more

January 25, 2021January 25, 2021

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

News, syndicated

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 ... Read more

January 21, 2021January 21, 2021

New CureDuchenne Initiative Provides Support, Education on COVID-19 Vaccination

News

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 ... Read more

January 20, 2021January 20, 2021

NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated

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 ... Read more

January 19, 2021January 19, 2021

Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial

News

A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with repeat dosing in a small clinical trial, according to a press release. The Phase 1/2 ... Read more

January 14, 2021January 14, 2021

Dosing Begins in Phase 3 Trial of Pfizer’s Gene Therapy for Duchenne

News

The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne muscular dystrophy (DMD). “The initiation of our pivotal trial, which is the ... Read more