While most exon-skipping tools target only one sequence ... "But for diseases like Alzheimer's, Parkinson's, Huntington's or Duchenne's muscular dystrophy, this approach holds a lot of potential.

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 ...

PPMD looks forward to working with Wave as they expediently bring WVE-N531 and their broader exon skipping pipeline to the Duchenne community.” All 11 participants in the trial have advanced to ...

(WCIA) — Researchers at the University of Illinois have found that a gene editing tool allows them to ... “With current ...

The delivery of therapeutic sequences for the treatment of inherited genetic diseases such as Duchenne muscular dystrophy (DMD ... Antisense oligonucleotides to the boundary sequences of exon and ...

Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon ...

Controversy has dogged Sarepta’s Duchenne muscular dystrophy therapy ... has published a draft report that not only suggests Sarepta’s exon-skipping drug is too expensive, but also that ...

Duchenne muscular dystrophy (DMD ... dose-escalation study of an oligomer that allows for exon skipping, restoring the open reading frame of the DMD gene and allowing a functional dystrophin ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest ...

ELEVATE-45 is the second of three novel exon skipping Duchenne programs the Company expects to progress into global clinical development in 2025 – ELEVATE-45-201 is a global, two-part ...

It is expected that the Duchenne Muscular Dystrophy market, in the 7 major markets, will reach $5.2 billion by 2033. This is a very large market opportunity, and there are other exon skipping ...