September 25, 2018
We’re pleased to share this message from our friends at Stand for Duchenne about a new initiative they have created called Reclaim the Day. Many Duchenne families, including ours, will never forget the day their children were diagnosed. The feeling of shock, panic, and helplessness is only a fraction of the emotions that we all […]
September 25, 2018
from Catabasis Pharmaceuticals
– Pivotal Study of Novel Inhibitor of NF-kB, a Key Driver of Skeletal Muscle Disease and Cardiomyopathy in Duchenne — CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy (DMD). Edasalonexent inhibits NF-kB, which is the key link […]
September 24, 2018
from Kate Sheridan
Sarepta’s early-stage clinical trial for a gene therapy for Duchenne muscular dystrophy is back on schedule, the Cambridge biotech said Monday. The Food and Drug Administration had put the trial on hold in July. The FDA ordered the hold after small fragments of DNA turned up in the treatment, which is being used at Nationwide […]
September 24, 2018
On Saturday, September 22 more than 100 people came out to the Jet Aircraft Museum in London, Ontario for the first “Pull a Plane to Defeat Duchenne”. This first-time event was held in partnership with Western University’s Commercial Aviation Management program. Participants teamed up to pull a 10,000lb, beautifully-restored Canadian T33 jet 50 metres across the […]
September 21, 2018
The Jesse’s Journey Board of Directors is pleased to announce the appointment of Jeff Preston, Ph.D. as Chair of the Board, effective September 17, 2018. Jeff has been a long-time supporter of Jesse’s Journey, having sat as a board member for the past two years. He is also active in fundraising as the inspiration behind […]
September 21, 2018
Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory […]
September 21, 2018
from Sarepta Therapeutics, Inc.
— Relying upon Scientific Advisory Group input, Sarepta will seek further scientific advice from European Medicines Agency on a possible path to bring eteplirsen to patients in Europe — CAMBRIDGE, Mass., September 21, 2018 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic […]
September 6, 2018
We have a new leader of the team here at Jesse’s Journey. We are pleased to announce that Perry Esler will be assuming the role of Executive Director on October 1st. Perry is no stranger to Jesse’s Journey, having been on the road with us in 1995. He comes to us with more than […]
September 4, 2018
from CBC Radio
Researchers used CRISPR gene editing successfully in a large animal to repair DNA mutations Listen Here A team of researchers may be one step closer to curing a devastating form of muscular dystrophy, after announcing they successfully used gene editing to stop the progression of the disease in dogs. “To say it was exhilarating to see the […]
August 31, 2018
from UT SOUTHWESTERN MEDICAL CENTER
DALLAS – Aug. 30, 2018 – Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline. The research published in Sciencedocuments unprecedented […]
