DMD

May 6, 2019

Gene Therapy Candidate GALGT2 Found Safe in Parent Project-Funded Toxicology Study

News

The investigational gene therapy GALGT2 for Duchenne muscular dystrophy (DMD) was found to be safe in a preclinical study conducted at the Research Institute at the Nationwide Children’s Hospital, ... Read more

April 12, 2019June 21, 2019

Single Dose of CRISPR Gene Therapy May Succeed as Long-term Treatment for DMD, Mouse Study Shows

Duchenne Muscular Dystrophy News, News

A single dose of gene therapy based on CRISPR-Cas9 may successfully treat Duchenne muscular dystrophy (DMD) in the long term or even permanently, a preclinical study in mice shows. The findings shed ... Read more

March 20, 2019April 5, 2019

New Enhanced Microdystrophin Versions May Help Restore Muscle Function in DMD, Mouse Study Suggests

News

Engineered small versions of dystrophin protein, known as microdystrophins, have shown the ability to enhance muscle strength and function in preclinical models of Duchenne muscular dystrophy (DMD). Researchers from ... Read more

March 6, 2019March 22, 2019

Raxone (Idebenone) Shows Promise as DMD Therapy in SYROS Trial

News

Long-term treatment with Raxone (idebenone) slows down loss of respiratory function in Duchenne muscular dystrophy (DMD) patients for up to six years, according to recent results from Santhera Pharmaceuticals’ SYROS trial. ... Read more

October 31, 2018October 31, 2018

Cure Duchenne Kicks Off Inaugural ‘Pinkies Up to CureDuchenne’ Fundraising Campaign

News

A campaign hopes to raise awareness of Duchenne muscular dystrophy (DMD) and promote research toward a cure by asking supporters to paint a nail on one of their little fingers ... Read more

October 1, 2018October 1, 2018

MuscleMap Automated Dystrophin Analysis Tool Can Help in DMD Assessment, Study Suggests

News

Flagship Biosciences has developed an automated method to analyze muscle fibers (myofibers) and quantify key disease-related biomarkers, such as dystrophin, for Duchenne muscular dystrophy (DMD). The algorithm, known as ... Read more

September 28, 2018October 26, 2018

Phase 3 Trial of Edasalonexent in Treating DMD Opening Soon, Catabasis Says

News

Catabasis Pharmaceuticals is launching a global Phase 3 trial to evaluate the potential of edasalonexent to treat boys with Duchenne muscular dystrophy (DMD) regardless of mutation type. The soon-to-open PolarisDMD trial is a ... Read more

August 24, 2018August 24, 2018

Canadian Collaboration Gives Funding Boost to Duchenne MD Research

News

AbCellera Biologics Inc. has announced a collaboration with Fabio Rossi, MD, PhD, and Michael Underhill, PhD, of the University of British Columbia, to develop therapeutic antibodies for the treatment ... Read more

May 7, 2018May 7, 2018

Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002

News

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants. The HOPE-2 ... Read more

April 20, 2018April 20, 2018

#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

News

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data ... Read more

Pin It on Pinterest