Muscular Dystrophy News

May 28, 2020May 28, 2020

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

NS Pharma’s lead candidate viltolarsen safely and effectively increases dystrophin levels and promotes improvements in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, final data from a Phase 2 trial show. “As a ... Read more

May 26, 2020May 26, 2020

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

Muscular Dystrophy News

A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability in boys with Duchenne muscular dystrophy (DMD), final study results show. ATL1102, developed by Antisense Therapeutics, was ... Read more

May 21, 2020May 21, 2020

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

Muscular Dystrophy News

The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid regimen to more effectively slow lung function decline in boys and men with Duchenne muscular ... Read more

May 19, 2020May 19, 2020

Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

Muscular Dystrophy News

Pfizer’s experimental gene therapy PF-06939926 promotes sustained production of a shorter but functional version of dystrophin — the protein lacking in Duchenne muscular dystrophy (DMD) — and improves motor abilities ... Read more

May 14, 2020May 14, 2020

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

Muscular Dystrophy News

One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac and upper limb function in boys and young men at advanced stages of the disease, ... Read more

May 12, 2020May 12, 2020

IGNITE DMD Trial Remains on Hold, FDA Requests More Gene Therapy Data

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) is requesting more information on the process used to manufacture the gene therapy SGT-001, as it reviews the clinical hold placed on the Phase 1/2 ... Read more

May 7, 2020May 7, 2020

Experts Offer Advice for Duchenne, Becker Patients Amid COVID-19 Pandemic

COVID-19 News, Muscular Dystrophy News

People with Duchenne and Becker muscular dystrophy should not forego their routine health assessments during the COVID-19 pandemic, but are encouraged to transition their care to telemedicine and to medical tests performed at home ... Read more

May 5, 2020May 5, 2020

The Power of Empathy Among Friends

Muscular Dystrophy News

I’ve never considered myself to be insecure about my disability. My use of a mobility aid has never dulled my social butterfly persona, except on a few occasions when I’ve ... Read more

May 5, 2020May 5, 2020

CureDuchenne Ventures Gives $1M to Support Non-viral Gene Therapy Work

Muscular Dystrophy News

CureDuchenne Ventures has given $1 million in seed funding to support the development of Myosana Therapeutics‘ potential non-viral gene therapy for people with Duchenne muscular dystrophy (DMD). The money is expected to advance ... Read more

April 30, 2020April 30, 2020

PTC and Sarepta Say MD Treatment Supplies Uninterrupted, Encourage Use of Patient Support Programs

Muscular Dystrophy News

Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a Wednesday webinar ... Read more

April 30, 2020April 30, 2020

The Impossibility of Putting a Value on a Life

Muscular Dystrophy News

Author’s note: The COVID-19 crisis has already lasted several months. The reality is that we don’t know how this will go. But as a community, my opinion is that we ... Read more

April 29, 2020May 8, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

COVID-19 News, Muscular Dystrophy News

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their ... Read more

April 28, 2020April 28, 2020

MDA’s Medical Advisory Team to Help Guide Research and Patient Care

Muscular Dystrophy News

The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related to research and clinical care in muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders. ... Read more

April 23, 2020May 8, 2020

COVID-19 Delays Phase 3 Trial of Givinostat in DMD, Italfarmaco Says in Webinar

COVID-19 News, Muscular Dystrophy News

Because of the global COVID-19 pandemic, Italfarmaco has paused enrollment of boys with Duchenne muscular dystrophy (DMD) in a Phase 3 trial of its investigational treatment givinostat, the company said ... Read more

April 21, 2020April 21, 2020

The ‘Crip Camp’ Utopia Made Us a Better Society Today

Muscular Dystrophy News

Netflix’s new documentary “Crip Camp” transports viewers to the peak era of peace-preaching hippies and civil rights activists. But amid these revolutions, another minority group struggled to find belonging: the ... Read more