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November 22, 2019November 22, 2019

Method Comparing RNA Differences Between Gene Copies May Help Find Cause of Disease, Study Suggests

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A new technique that compares RNA production between an individual’s two gene copies may help find genetic alterations that lead to muscular dystrophy and other rare diseases, a study ... Read more

November 20, 2019November 20, 2019

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

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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 18, 2019November 18, 2019

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

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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 15, 2019November 15, 2019

PPMD Revamps Duchenne Registry with New App

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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

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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 13, 2019November 13, 2019

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

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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

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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 9, 2019November 9, 2019

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

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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

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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

November 6, 2019November 6, 2019

Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests

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The investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root cause of muscle degeneration in facioscapulohumeral muscular dystrophy (FSHD), new research ... Read more

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