Click here for the recorded webinar: https://cc.readytalk.com/cc/playback/Playback.do?id=2lyea5 CureDuchenne has been an early and important funder of exon skipping research for Duchenne. As we gain visibility to an actual therapeutic with exon […]
CureDuchenne was pleased to host the “New Duplication Exon 2 Mouse Model: A Tool for Studies of Duplication Skipping for Duchenne” a webinar with Dr. Kevin Flanigan on November 21, 2013. The webinar was an opportunity for parents to learn about the duplication mutation research and an exciting exon skipping method using AAV delivery with U7 from Dr. Kevin Flanigan at Nationwide Children’s Hospital. CureDuchenne has provided the lead funding for Dr. Flanigan’s duplication research.
Nine-year-old Tyler Armstrong of Yorba Linda, Calif., will attempt a record breaking climb of Mt. Aconcagua in Argentina, the highest peak (22,837 ft.) in both the Western and Southern Hemispheres, to benefit CureDuchenne
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will host a Ì¢âÂÒNew Duplication Exon 2 Mouse Model: A Tool for Studies of Duplication Skipping for DuchenneÌ¢âÂÌ_ webinar with Dr. Kevin Flanigan from Nationwide ChildrenÌ¢âÂã¢s Hospital on Thursday, November 21 at noon EST/9:00 a.m. PST. The webinar is an opportunity for the Duchenne community to learn about the duplication mutation research and an exciting exon skipping method using AAV delivery with U7. CureDuchenne has provided the lead funding for Dr. FlaniganÌ¢âÂã¢s duplication research.
CureDuchenne, a national nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, announced today that actor Josh Duhamel is featured in a new public service announcement (PSA) on behalf of the organization.
Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (‘DMD’) and C. difficile infection, announces that its Phase 1b Clinical Trial Application for SMT C1100 has received approval from the UK Medicines and Healthcare products Regulatory Agency (‘MHRA’) and the Ethics Review Committee. SMT C1100 is a small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying genetic fault.
Walking ability improved markedly in three of the first four patients with Becker muscular dystrophy to receive a gene therapy designed to promote muscle growth, a researcher said here.
Scientists in The Research Institute at Nationwide Children’s Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.
