April 6, 2020April 2, 2020
Self-quarantine Has Leveled the Social Playing Field
columns, Hawk's-Eye View - a Column by Hawken Miller
I’ve always sought silver linings, including in this season of social distancing. Many people now exclusively use technology to ... Read more
April 2, 2020April 6, 2020
NORD’s Advice on COVID-19: You’re Stronger Than You Think
News, syndicated
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you ... Read more
April 2, 2020April 2, 2020
MDA Hosting Online Q&A on COVID-19 Today
News
The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people ... Read more
March 31, 2020March 31, 2020
Trial Use of Edasalonexent Not Likely to Raise COVID-19 Risk, Catabasis Reports
News
Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune ... Read more
March 27, 2020March 30, 2020
Make the Most Out of Your Time – Lockdown Tips
Social Clips
Our Forums moderator Kevin Schaefer shared great advice for making the most out of your time while at home. To ... Read more
March 26, 2020March 24, 2020
The Muscular Dystrophy Community Counts on Accessibility Improvements
columns, Living, Learning, Thriving - a Column by Ralph Yaniz
I feel strange not writing about the pandemic crisis in this column, but we’re already bombarded with information about ... Read more
March 26, 2020March 26, 2020
Phase 1 Results Support Losmapimod as FSHD Treatment
News
Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as ... Read more
March 26, 2020March 26, 2020
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
News
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been ... Read more
March 24, 2020March 24, 2020
Sarepta Working to Ensure Treatment Access, Clinical Trials Continuity Amid COVID-19 Pandemic
News
Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), ... Read more
March 23, 2020March 23, 2020
Emflaza Supply Not Affected by COVID-19 Pandemic, PTC Therapeutics Says
News
The supply of Emflaza (deflazacort), a treatment for people with Duchenne muscular dystrophy (DMD), will not be ... Read more
March 20, 2020March 20, 2020
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
News
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy ... Read more
March 20, 2020March 20, 2020
Viltolarsen Available to U.S. DMD Patients Under Expanded Access Program
News
NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy (DMD) patients to gain access to ... Read more

April 6, 2020April 2, 2020

Self-quarantine Has Leveled the Social Playing Field

columns, Hawk's-Eye View - a Column by Hawken Miller

I’ve always sought silver linings, including in this season of social distancing. Many people now exclusively use technology to communicate with family, friends, and co-workers to reduce viral spread. ... Read more

April 2, 2020April 6, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News, syndicated

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most ... Read more

April 2, 2020April 2, 2020

MDA Hosting Online Q&A on COVID-19 Today

News

The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. ... Read more

March 31, 2020March 31, 2020

Trial Use of Edasalonexent Not Likely to Raise COVID-19 Risk, Catabasis Reports

News

Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune system’s ability to work as it should, making it unlikely to ... Read more

March 26, 2020March 24, 2020

The Muscular Dystrophy Community Counts on Accessibility Improvements

columns, Living, Learning, Thriving - a Column by Ralph Yaniz

I feel strange not writing about the pandemic crisis in this column, but we’re already bombarded with information about it, and it appears we will deal with these issues ... Read more

March 26, 2020March 26, 2020

Phase 1 Results Support Losmapimod as FSHD Treatment

News

Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest its effectiveness as a treatment for facioscapulohumeral muscular dystrophy (FSHD). The data, “Phase 1 ... Read more

March 26, 2020March 26, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more

March 24, 2020March 24, 2020

Sarepta Working to Ensure Treatment Access, Clinical Trials Continuity Amid COVID-19 Pandemic

News

Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen), both approved for the treatment of Duchenne muscular dystrophy (DMD), during the ... Read more

March 23, 2020March 23, 2020

Emflaza Supply Not Affected by COVID-19 Pandemic, PTC Therapeutics Says

News

The supply of Emflaza (deflazacort), a treatment for people with Duchenne muscular dystrophy (DMD), will not be affected by the COVID-19 pandemic, according to PTC Therapeutics, its developer. In ... Read more

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more

March 20, 2020March 20, 2020

Viltolarsen Available to U.S. DMD Patients Under Expanded Access Program

News

NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy (DMD) patients to gain access to its investigational exon-skipping therapy viltolarsen. The program will allow physicians to prescribe ... Read more