Tag: Exon skipping

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Recent Related Articles to Exon skipping

As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators

Added to Collection on: 2022-06-26 14:41:00

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Muscular dystrophy
Branches of biology
Life Sciences
Biology
Duchenne muscular dystrophy
Becker muscular dystrophy
Sarepta Therapeutics
Dystrophin
Eteplirsen
Placebo
Biostrophin
Exon skipping
Italfarmaco Group

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Sarepta looks for quick approval for Duchenne muscular dystrophy gene therapy treatment

Added to Collection on: 2022-07-29 14:15:46

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Muscular dystrophy
Branches of biology
Life Sciences
Duchenne muscular dystrophy
Sarepta Therapeutics
Health sciences
Dystrophin
Dystrophy
DMD
Ezutromid
Exon skipping
Sarepta Therapeutics Inc.

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Avidity Biosciences Announces Phase 1/2 EXPLORE44™ Trial of AOC 1044 for Duchenne ...

Added to Collection on: 2022-10-12 09:17:09

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Muscular dystrophy
Branches of biology
Biology
Duchenne muscular dystrophy
Exon skipping
Dystrophin
Facioscapulohumeral muscular dystrophy
Morpholino
Dystrophy
Draft:CureDuchenne
Drisapersen
Avidity Biosciences Inc.
Duchenne
SAN DIEGO

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WVE-N531's Impact on Exon Skipping, PROs Improved With Early DMT Initiation, Carbon ...

Added to Collection on: 2022-12-24 12:08:16

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RTT
Branches of biology
Amines
Sleep disorders
Muscular dystrophy
Psychiatric diagnosis
Entheogens
N,N-Dimethyltryptamine
Duchenne muscular dystrophy
Insomnia
Exon skipping

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Sarepta's muscular dystrophy therapy fails to meet main goal in late-stage trial | Reuters

Added to Collection on: 2023-10-30 21:52:01

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Muscular dystrophy
Duchenne muscular dystrophy
Sarepta Therapeutics
Gene therapy
Dystrophin
Delandistrogene moxeparvovec
Therapy
DMD
Exon skipping
Drisapersen
SRPT.O

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FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease

Added to Collection on: 2024-03-22 16:51:13

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Biotechnology
Muscular dystrophy
Rare diseases
Genetic engineering
Duchenne muscular dystrophy
Applied genetics
Deflazacort
Delandistrogene moxeparvovec
Gene therapy
PTC Therapeutics
Exon skipping
Sarepta Therapeutics

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Gene Therapy GNT0004 Demonstrates Early Efficacy, Safety in Duchenne Muscular Dystrophy

Added to Collection on: 2024-06-12 22:08:07

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Muscular dystrophy
Biotechnology
Rare diseases
Genetic engineering
Duchenne muscular dystrophy
Delandistrogene moxeparvovec
Dystrophin-associated protein complex
Sarepta Therapeutics
Gene therapy
Dystrophin
Exon skipping
Steve Wilton
Duchenne

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New Phase 1/2 EXPLORE44 Trial Data Shows Continued Treatment Effect of AOC 1044 in ...

Added to Collection on: 2024-08-13 18:31:56

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Muscular dystrophy
Duchenne muscular dystrophy
Exon skipping
Dystrophin
Creatine
Steve Wilton
Drisapersen
Duchenne
Michael Kelly
Diana Castro

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FDA Gives Approval For Duchenne Muscular Dystrophy Pediatric Treatment | Science 2.0

Added to Collection on: 2024-09-11 19:02:01

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Muscular dystrophy
Duchenne muscular dystrophy
Dystrophin
Exon skipping
Steve Wilton
Nippon Shinyaku Co. Ltd.

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Surfing on new Duchenne data, Wave charges $175m offering | pharmaphorum

Added to Collection on: 2024-09-25 11:17:25

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Muscular dystrophy
Dystrophin
Duchenne muscular dystrophy
DMD
Viltolarsen
Exon skipping
Dystrophin-associated protein complex

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