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 ...

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

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

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 ...

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 ...

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

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 ...

As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its Duchenne muscular ... trial of Wave’s exon skipping oligonucleotide ...

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 ...