January 22, 2020
Santhera Pharmaceuticals have shared their January update. “Our main focus in the U.S. in the first half of the year is to bring the SIDEROS clinical trial to full enrollment and we ask the community’s help, now more than ever, to achieve this goal. The SIDEROS clinical trial, which investigates the safety and efficacy […]
December 24, 2019
Sarepta Therapeutics Announces Partnership with Roche in Territories Outside the United States for its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy, SRP-9001 “…Sarepta and Roche have entered into a licensing agreement that would provide Roche exclusive rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) outside of the United States. As a mission-driven organization committed to families impacted by […]
November 29, 2019
Jesse’s Journey is proud to announce its gift of $300,000 towards a breakthrough clinical trial, which may empower local researchers to discover a therapeutic solution to the degenerative disease Duchenne muscular dystrophy. The clinical trial is building upon promising Phase I research involving myoblast transplantation. This therapeutic approach applies to all Duchenne patients, independently of […]
November 12, 2019
More than 75 Canadians came together for the inaugural Defeat Duchenne Family Forum presented by PTC Therapeutics on Saturday, November 2, 2019, in Calgary, Alberta. Families from across Western Canada affected by Duchenne muscular dystrophy came together with world-renowned researchers, clinicians, and industry professionals for a day of education, inspiration, and hope. Read more here.
November 12, 2019
Unless you’ve experienced it for yourself, it’s nearly impossible to understand the ramifications of finding out your loved one has Duchenne muscular dystrophy. Jesse’s Journey is lucky to have the support of families who are brave enough to share their stories because they know it’s the only way we can hope to inspire understanding and […]
October 24, 2019
from Joana Carvalho, MSc
Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD) being developed by ReveraGen Biopharma, continues to improve muscle function in DMD patients with fewer side effects than standard corticosteroids, topline data in 23 boys in an ongoing Phase 2 trial show. These findings were detailed in a late-breaking presentation by Eric Hoffman, PhD, chief executive […]
October 21, 2019
from Matt Hoffman
Phase 2 study results of edasalonexent, an investigational oral small molecule NF-kB inhibitor for the treatment of Duchenne muscular dystrophy (DMD), suggest that the therapy is associated with slowed disease progression, as well as a good safety profile over the course of more than 50 patient-years of exposure in boys with the disease.1 Treatment with […]
May 28, 2019
With over 7,000 rare conditions already identified and dozens more being discovered every day, it is estimated that rare diseases impact 1 in 12 Canadians, two-thirds of whom are children. But each disease affects only a handful of individuals, so understanding and medical expertise are limited and uneven across the country. Many of these conditions […]
May 24, 2019
from ir.ptcbio.com
– $545,000 awarded to help improve Duchenne muscular dystrophy diagnosis and screening across the globe –SOUTH PLAINFIELD, N.J., May 20, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the three funding recipients from PRIORITY, an annual program to support outstanding clinical research by the rare genetic disorders community. This year’s funding will accelerate innovative research projects to improve Duchenne […]
May 14, 2019
from Newswire.ca
Government of Canada consults on new approach to fast-track the review of treatments for serious, rare and debilitating diseases OTTAWA, May 13, 2019 /CNW/ – The Government of Canada is committed to helping all Canadians access affordable treatment options they need to help them improve and maintain their health. For many Canadians, managing the symptoms of rare or debilitating diseases can be […]
