News

February 19, 2021February 19, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 18, 2021February 18, 2021

MDA Launches 39th Annual #Shamrocks Fundraiser

News

The Muscular Dystrophy Association (MDA) has launched its 39th annual #MDAShamrocks campaign — the largest St. Patrick’s fundraiser in the U.S. — aiming to raise money and awareness about muscular ... Read more

February 16, 2021February 16, 2021

Deleting Mss51 Gene Reduces Fatigue, Increases Endurance in DMD Mice

News

By deleting the Mss51 gene, scientists were able to reduce fatigue, increase endurance, and ease mitochondrial impairment in a mouse model of Duchenne muscular dystrophy (DMD). Mss51 is a gene ... Read more

February 11, 2021February 11, 2021

PPMD Receives Award to Improve Neurobehavioral Care in Duchenne

News

Parent Project Muscular Dystrophy (PPMD) has received an award of more than $100,000 from the Duchenne Research Fund to address neurobehavioral care in people with Duchenne muscular dystrophy (DMD). “We are ... Read more

February 11, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

News, syndicated

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 ... Read more

February 9, 2021February 9, 2021

Translarna Increased Dystrophin Levels in Phase 2 DMD Study

News

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 ... Read more

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 Duchenne ... 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 costs ... 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 revamped, ... 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, was ... Read more