2017, November 17th
On a regular basis, team La Force will relay breaking news to the DMD community in News! our latest blog. We provide news highlights to give you quick-and-easy access to the most recent information about what’s happening worldwide in DMD advocacy and, more importantly, advances in therapy and the availability of new treatments. The short news alerts will also appear in our newsletter and, for members of our DMD community, on the Sign-in page.
The DMD News helps you to stay informed, become aware and share what’s happening in the DMD community to bring us closer to a cure.
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Jesse’s Journey & Max’s Big Ride fund STAT3 inhibitor research
On October 31, 2017, Jesse’s Journey and Max’s Big Ride – two non-profit groups founded by parents of children with Duchenne muscular dystrophy (DMD) – presented $336,000 in funding to Dr. Patrick Gunning, Professor of Chemistry, Canada Research Chair in Medicinal Chemistry Tier II, and head of the Gunning Group, a research laboratory at the University of Toronto Mississauga.
Max’s Big Ride raised $35,000 for Max’s Big Fellowship, a new Ph.D. fellowship at the Gunning Group. It was awarded to Yasir Raouf, who will study STAT3 inhibitors in DMD. Earlier this year, Jesse’s Journey approved a $300,000 grant to Dr. Gunning to fund an exciting, first-time, preclinical study of STAT3 and other protein inhibitors to treat DMD.
For more information about the study and how you can get involved: read here
Duke University and Sarepta hunt for gene-editing therapy
Muscular Dystrophy News Today reports that Sarepta Therapeutics has teamed up with Dr. Charles Gersbach’s biomedical engineering laboratory at Duke University to develop gene-editing therapies for Duchenne muscular dystrophy (DMD).
Dr. Gersbach is a pioneer in the use of CRISPR/Cas9 technology, a gene-editing tool that can remove abnormal parts of the dystrophin gene. Mutations in this gene cause DMD. Gerbach says that cutting out specific exons (regions that contain the genetic code or “blueprint” for the dystrophin protein) “has the potential to correct a majority of DMD mutations.”
His team has already shown, in mice models of DMD, that removing abnormal regions of the dystrophin gene restores muscle function. The edited gene was able to produce a normally functioning protein that improved muscle strength in mice.
For more information: read here
New clinical trial of exon-51 skipping therapy for DMD
Wave Life Sciences has begun the first clinical trial of WVE-210201, an exon 51-skipping therapy, in patients with Duchenne muscular dystrophy (DMD). The multicenter, double-blinded, placebo-controlled, dose-finding, Phase 1 clinical trial will study which intravenous doses of WVE-210201 are safe and tolerable in a subgroup of patients with DMD. About 13% of patients with DMD have exon 51 mutations in the dystrophin gene.
The study will enroll up to 40 patients between 5 and 18 years of age, either able or unable to walk. It will begin in the USA, then expand to Europe and other regions. Results are expected in late 2018.
WVE-210201 is an antisense oligonucleotide that throws a cog in the wheels of protein manufacturing. It forces the cellular machinery to skip over a damaged exon – a specific section of genetic code – then resume regular “reading” of the protein’s “blueprint.” The exon 51-skipping process produces an incomplete but functional dystrophin protein, which may restore some muscle function in patients with this mutation.
For more information: read here
