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myotonic muscular dystrophy
PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1
BioSpace
Tue, 02/20/24 - 05:49 pm
PepGen
oligonucleotides
PGN-EDODM1
myotonic muscular dystrophy
Dyne's shares surge after promising data on muscle disorder treatment
Reuters
Wed, 01/3/24 - 09:33 am
Dyne Therapeutics
clinial trials
DYNE-101
myotonic muscular dystrophy
Duchenne Muscular Dystrophy
PepGen cleared by FDA to begin study of muscular dystrophy drug
BioPharma Dive
Thu, 10/12/23 - 05:20 pm
PepGen
FDA
myotonic muscular dystrophy
Awaiting FDA hold letter, PepGen accepts 2nd oligonucleotide is no closer to clinic
Fierce Biotech
Wed, 06/14/23 - 11:30 am
PepGen
oligonucleotide
FDA
myotonic muscular dystrophy
PGN-EDODM1
Avidity unveils positive topline data from DM1 therapy trial
Clinical Trials Arena
Fri, 04/28/23 - 10:10 am
Avidity Biosciences
AOC 1001
clinical trials
myotonic muscular dystrophy
Fresh from Vertex payday, Entrada's DMD plans are derailed by FDA hold
Fierce Biotech
Tue, 12/20/22 - 10:42 am
Entrada Therapeutics
Vertex Pharmaceuticals
FDA
clinical trials
Duchenne Muscular Dystrophy
myotonic muscular dystrophy
ENTR-601-44
Avidity Biosciences Stock Soars On New RNA Data
BioSpace
Wed, 12/14/22 - 07:21 pm
Avidity Biosciences
RNA
AOC-1001
myotonic muscular dystrophy
Vertex pays Entrada $224M for preclinical rare disease drug, opening new front in assault on dystrophy
Fierce Biotech
Thu, 12/8/22 - 10:35 am
Vertex Pharmaceuticals
Entrada Therapeutics
ENTR-701
myotonic muscular dystrophy
With Secreted Proteins Map, Juvena Finds Path to $41M for New Regenerative Meds
MedCity News
Tue, 11/8/22 - 10:20 pm
Juvena Therapeutics
myotonic muscular dystrophy
Sanofi, miRecule Ink Muscular Dystrophy Research Deal Worth up to $430M
BioSpace
Tue, 10/4/22 - 11:43 pm
Sanofi
miRecule
myotonic muscular dystrophy
FDA Hits Avidity's AOC for Myotonic Dystrophy Type 1 with Partial Hold
BioSpace
Wed, 09/28/22 - 10:38 am
Avidity Biosciences
AOC 1001
myotonic muscular dystrophy
FDA
clinical trials
Design Therapeutics launches with $45m to develop a new class of disease-modifying therapies for serious degenerative disorders
Pharmaceutical Business Review
Mon, 03/23/20 - 11:36 am
Design Therapeutics
degenerative diseases
Fragile X syndrome
myotonic muscular dystrophy
Eli Lilly-backed biotech grabs $100M to dispatch antibody-oligonucleotide conjugates after muscular dystrophy
Endpoints
Wed, 11/13/19 - 10:09 am
myotonic muscular dystrophy
Eli Lilly
R&D
Avidity