myotonic dystrophy Type 1

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Research

RNA-targeting drug relaxes muscles in myotonic dystrophy mice

Arrakis’ small molecule was also able to restore normal RNA splicing in muscle cells derived from patients.
Darren Incorvaia Dec 4, 2024 7:00am
Novartis

Novartis signs $1.1B deal for muscular dystrophy biotech

Nov 21, 2024 8:19am
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PepGen gains a little pep in its step as FDA clears trial hold

Oct 12, 2023 7:00am
Letter opening FDA clinical hold

Awaiting FDA letter, PepGen oligonucleotide no closer to clinic

Jun 14, 2023 9:05am
Stop sign

FDA blocks PepGen's attempt to get 2nd oligonucleotide to clinic

May 31, 2023 8:11am
Photo of a chain being held together by a piece of rope

Avidity says FDA has 'eased' partial hold on lead asset

May 17, 2023 11:16am