News

July 2, 2020July 2, 2020

Coronavirus Relief Fund Created to Help DMD Patients, Families

News

The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and tightened budgets. But for families affected by Duchenne muscular dystrophy (DMD), who front extra ... Read more

June 30, 2020June 30, 2020

CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk

News

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne ... Read more

June 25, 2020June 25, 2020

Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer

News

A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has dosed its first healthy volunteer, ML Bio Solutions announced. The company also ... Read more

June 23, 2020June 23, 2020

Collaboration Provides Cough Assist Machines to British Columbia Residents With Neuromuscular Disease

News

A partnership between Muscular Dystrophy Canada (MDC) and Technology for Living (TFL) will seek to provide a cough assist device to any adult in British Columbia who has neuromuscular disease ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

cystic fibrosis, News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 18, 2020June 18, 2020

Sarepta’s Gene Therapy for DMD Showing to Safety, Efficacy at One Year

News

At the one year mark, Sarepta Therapeutics’ SRP-9001 micro-dystrophin gene therapy continues to show positive results in four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 study. ... Read more

June 16, 2020June 16, 2020

MDA Holds Q&A on Navigating COVID-19, and Area Reopenings, Safely

News

People with neuromuscular disorders such as muscular dystrophy are not at a higher risk of acquiring COVID-19, but they do face greater challenges should they become infected. Creating tailored ... Read more

June 11, 2020June 11, 2020

Viltolarsen Earns European Orphan Drug Designation as DMD Treatment

News

The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, Nippon Shinyaku announced in a press release. The ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... Read more

June 9, 2020June 15, 2020

SRP-9003 Gene Therapy for LGMD2E Shows Benefits After 1 Year

News

A single administration of the gene therapy SRP-9003 at a low dose continues to lead to significant improvements in functional measures in three children with limb girdle muscular dystrophy ... Read more

Pin It on Pinterest