Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

Phil Lambert, Ph.D., chief scientific officer of Satellos Bioscience Inc., explains the mechanism of action of the company's experimental Duchenne muscular dystrophy drug, SAT-3247, and the thinking ...

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a ... with a gene mutation that can be treated by skipping exon 45 (a certain section of genetic code), doctors can prescribe the ...

Certain genetic mutations in patients with DMD influence the timing of ambulation loss, with exon 44 skipping ... phenotype correlations in Duchenne muscular dystrophy (DMD).

About 60 to 70 percent of variants in patients with Duchenne muscular dystrophy are deletions, while 5 to 15 percent are duplications, and 20 percent are single nucleotide variants or other small ...

Duchenne muscular dystrophy (DMD ... be used if you have specific types of genetic mutations, including any deletion of exon 8 or exon 9 in the DMD gene. Your health care provider may test ...

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug ... the dystrophin gene amenable to exon 51 skipping, the drug could ...

Duchenne muscular dystrophy (DMD ... is a promising tool to induce a gigantic genomic deletion and restore dystrophin protein via multi-exon skipping induction," says senior author Akitsu Hotta ...

Two index cases with both Duchenne muscular dystrophy and autism spectrum disorder ... In addition, five of the eight boys showed deletion in the dystrophin gene on polymerase chain reaction.

a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. The drug – formerly known as SRP-5051 – is a more potent version of Exondys 51 (eteplirsen), an exon-skipping therapy ...

Elevidys is approved for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon ... in advancing the treatment of Duchenne muscular dystrophy through innovative ...

Opens in a new tab or window Eight therapies are approved for Duchenne muscular dystrophy (DMD ... a nonsteroidal anti-inflammatory drug, exon-skipping anti-sense oligonucleotides, and an ...