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).

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 ...

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 ...

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 ...

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

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy ... DMD mutations amenable to exon 44 skipping (DMD44), a group ...

Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from ...

Duchenne muscular dystrophy has seen more progress in the ... This includes questions about the ability to combine gene therapy with exon-skipping treatments for those patients that are amenable.

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...

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 ...