November 20, 2019November 20, 2019

Long-term Treatment with Puldysa Reduces Lung Function Decline in DMD Patients, Study Shows

News

Long-term treatment with Puldysa (idebenone) provides a stable reduction of lung function decline by approximately 50% in people with Duchenne muscular dystrophy (DMD), a study shows. The research, “Long-term ... Read more

November 19, 2019November 18, 2019

Improving My Digestion with Enzyme Supplements

columns, You, Me and MD - a Column by Leah Leilani

It’s over a month since my gallbladder surgery, and I couldn’t be happier with my healing process. I previously wrote about adjusting to life after gallbladder removal and mentioned ... Read more

November 18, 2019November 18, 2019

NS Pharma to Provide Update on its Investigational Therapy Viltolarsen in Webinar

News

NS Pharma will share future plans for viltolarsen, its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) in a webinar hosted by Parent Project Muscular Dystrophy (PPMD) on Wednesday. The ... Read more

November 18, 2019November 14, 2019

Expressing an Attitude of Thankfulness This Holiday

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

When I pray, I like to start by thanking God for all of the blessings he has bestowed on me. It reminds me of how much I have to ... Read more

November 15, 2019November 15, 2019

PPMD Revamps Duchenne Registry with New App

News

Parent Project Muscular Dystrophy (PPMD) announced it is updating its global registry with a new, user-friendly app intended to empower people with Duchenne muscular dystrophy and their families to become ... Read more

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

November 14, 2019November 12, 2019

Let’s Stay on the Cutting Edge of Research

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

I can’t believe it’s been 13 years since I started exhibiting muscle loss. A lot has happened since then. My loss is slow but steady. Even when I plateau, ... Read more

November 13, 2019November 13, 2019

FDA Puts 2nd Clinical Hold on IGNITE DMD Trial of Gene Therapy SGT-001 Due to Patient Reaction

News

Owing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another clinical hold on a Phase 1/2 clinical trial of SGT-001, ... Read more

November 11, 2019November 13, 2019

Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued

News

The clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with Duchenne muscular dystrophy (DMD) has been discontinued, according to a letter to the DMD community by ... Read more

November 11, 2019November 8, 2019

Attack of the Bloodthirsty Mosquitoes

columns, You, Me and MD - a Column by Leah Leilani

Fall and winter have always been my favorite seasons. I eagerly await the cooler weather by prematurely washing my sweaters and drinking warm beverages. This year, I’m particularly excited ... Read more

November 9, 2019November 9, 2019

Local November 9th MDA Disease Education Events Span East, West Coast

News

The Muscular Dystrophy Association’s (MDA) efforts to support and advocate for a wide range of neuromuscular diseases are felt by patient communities nationwide. While the MDA’s scope is indeed ... Read more

November 8, 2019November 8, 2019

Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says

News

Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with Duchenne muscular dystrophy (DMD), a ... Read more