Multiple companies are digging in to add the next generation of exon skippers, gene therapies and medicines targeting more than skeletal muscle to their DMD pipelines, and for Michael Kelly, chief ...

Some types of muscular dystrophy can be fatal. Certain types, including Duchenne muscular dystrophy, can lead to problems with the heart and muscles used for breathing. Some people with MD may develop ...

The SALSA MC002 SMA Newborn Screen uses PCR amplification and melt curve analysis to determine the presence or absence of exon 7 in the SMN1 and SMN2 genes from a dried blood spot sample.

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TEMPE, Ariz., Jan. 16, 2025 /PRNewswire/ -- DMD Systems Recovery, LLC (DMD), a leader in Reuse First™ IT Asset Disposition (ITAD) services and a Certified B Corporation, has been ranked #27 ...

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

for patients with disease-causing mutations in the DMD gene amenable to exon 51 skipping. Dyne previously reported DELIVER trial data that showed the therapy led to increases in dystrophin levels and ...

Nay Maps is a South African actor born on 30th March 1989. He is the firstborn of three children born to the late Bishop Simeon Maphalala and his wife. Maps grew up alongside his two younger sisters ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...

For the small number of DMD patients with a gene mutation that can be treated by skipping exon 45 (a certain section of genetic code), doctors can prescribe the injectable drug casimersen (Amondys ...