welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans.
Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and to provide the seed funding necessary to accelerate his work in this company, we could not be more thrilled. This is a true testament to the power of venture philanthropy and I want to thank each and every donor and supporter who has believed in our work, and we are grateful to Dr. Olson for his dedication to Duchenne patients. Although we still have a long way to go before we confirm this therapy will be safe and effective in humans, this dramatic dystrophin restoration offers hope to all families affected by this disease.
rareRelated
-
Rimeporide in Patients With Duchenne Muscular DystrophyIn Duchenne Muscular Dystrophy (DMD) the...
-
Neurology care, diagnostics, and emerging therapies of the patient with Duchenne muscular dystrophyDuchenne muscular dystrophy is the most ...
-
Realizing the promise of gene therapy through collaboration and partnering: Pfizer’s viewGene therapy is a promising approach to ...
-
MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dys...NF-κB is activated from infancy in DMD,...
-
From Flask to Vat: The Challenges of Scaling Up Gene TherapyOver the past two decades, gene therapy ...
-
A Study of Tadalafil for Duchenne Muscular DystrophyThe main purpose of this study is to det...
-
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular DystrophyThe purpose of the study is to see wheth...
-
Santhera Provides Update on Filing for Conditional Marketing Authorization in Europe for Puldysa® (Idebenone) in Du...Santhera Pharmaceuticals announces its i...
