News & Updates | Duchenne Muscular Dystrophy

News & Resources

Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial

February 19, 2019

Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (DMD) patients show low levels of microdystrophin in patients’ muscles after three months of treatment with an initial low dose and continued safety, Solid Bioscience, the therapy’s developer, reported. Continued safety was also evident, the company said in a press release. It added […]

Capricor Resumes Dosing of Enrolled Patients in HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy

February 14, 2019

from Global News Wire

LOS ANGELES, Feb. 06, 2019 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a clinical-stage biotechnology company, today announced that it has resumed per protocol dosing of patients already enrolled in its HOPE-2 clinical trial of CAP-1002, the company’s novel cell therapy candidate to treat Duchenne muscular dystrophy. Approximately 20 young men and boys in advanced stages of […]

Discovery points to innovative new way to treat Duchenne muscular dystrophy

February 4, 2019

from Medical Xpress

Researchers at The Ottawa Hospital and the University of Ottawa have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. As reported in Cell Stem Cell, the team restored muscle stem cell function that is impaired in Duchenne muscular dystophy, resulting in efficient […]

International Preferences for Duchenne Treatments Study

January 29, 2019

The International Preferences for Duchenne Treatment Study is now open in Canada. Jesse’s Journey has partnered with Johns Hopkins University, Parent Project Muscular Dystrophy, and other Duchenne advocacy organizations from around the world on this important study. This survey is the first international study to measure the preferences and priorities of people with Duchenne and […]

Researchers overcome hurdle in CRISPR gene editing for muscular dystrophy

January 9, 2019

from EurekAlert

The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions. A research team led by Dongsheng Duan, PhD, at the University of Missouri School of Medicine has identified and overcome a barrier in CRISPR gene editing […]

Jesse’s Journey Grants 10 Millionth Dollar to Research

November 22, 2018

On November 21, Jesse’s Journey announced to donors, volunteers, and families who are facing Duchenne muscular dystrophy that as of 2018, Jesse’s Journey has granted a cumulative total of more than $10 million to research. For more than 20 years, Jesse’s Journey has been dedicated to funding research focused on Duchenne muscular dystrophy – the […]

Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program

January 4, 2019

from Global News Wire

CAMBRIDGE, Mass., Jan. 03, 2019 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne muscular dystrophy (DMD) clinical program has been selected for the U.S. Food and […]

Project Hercules Enlists Industries Help to Make Duchenne Therapies Affordable

January 4, 2019

from Muscular Dystrophy News Today

Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the day she learned her son, Eli, had Duchenne muscular dystrophy (DMD). “The initial diagnosis was so devastating. There’s no escape. It’s absolutely relentless,” she said. “When […]

Catabasis Pharmaceuticals Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy Progress Update and Additional Trial Sites Open for Enrollment

January 3, 2019

from Catabasis

— Patient Enrollment Underway in U.S. with Nine Locations Open for Enrollment — — Global Start-Up Activities Ongoing with Clinical Trial Application Approvals Received in Canada and Multiple European Countries — CAMBRIDGE, Mass., January 3, 2019 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared an update on clinical trial sites in the United […]

Potential DMD Therapy WVE-210201 Shows Positive Phase 1 Safety Results

December 27, 2018

from Muscular Dystrophy News Today

WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD), Wave Life Sciences announced. According to Wave Life Sciences, the positive safety data supports moving the therapy into a Phase 2/3 trial, planned to start in 2019, to confirm the safety profile and establish efficacy. The company […]

US drug developer Capricor places Duchenne drug trial on hold

December 27, 2018

from Channel NewsAsia

REUTERS: Capricor Therapeutics Inc said on Wednesday it had put on hold a clinical trial of its drug to treat Duchenne muscular dystrophy (DMD), a muscle-wasting disorder, citing a safety review. The review follows a severe allergic reaction during infusion of the drug, the company said in a filing. The patient responded well to medical […]

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