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December 4, 2019December 4, 2019

Avidity Secures $100M Funding to Advance Treatments for Myotonic Dystrophy, DMD

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Avidity Biosciences has obtained $100 million to advance its programs for muscle disorder therapies, including a potential Antibody Oligonucleotide Conjugates (AOC) treatment for myotonic dystrophy type 1 (MD1). Based on ... Read more

December 2, 2019December 2, 2019

New DMD Mouse Model Helps Monitor Real-Time Dystrophin Production, Study Reports

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A new mouse model of Duchenne muscular dystrophy (DMD) can help researchers test potential gene editing therapies by showing real-time levels of the dystrophin protein in muscles and the ... Read more

November 27, 2019November 27, 2019

Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy

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A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at University of Florida (UF). In ... Read more

November 25, 2019November 25, 2019

CureDuchenne Develops Biobank to Aid Duchenne Lab Research

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CureDuchenne has begun taking blood samples and skin biopsies to facilitate research on new treatments for Duchenne muscular dystrophy. The CureDuchenne Biobank is a partnership involving neurologist Tahseen Mozaffar, ... Read more

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

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