Tag: Exon skipping

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Recent Related Articles to Exon skipping

PepGen to Present a CureDuchenne Webinar on Unlocking the Potential of Exon Skipping ...

Added to Collection on: 2021-04-19 12:00:00

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Muscular dystrophy
Branches of biology
Duchenne muscular dystrophy
Neuromuscular disease
Exon skipping
Dystrophy
Dystrophin
Oligonucleotide
DMD
Prosensa
PepGen Inc.

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Exon Skipping Therapies May Be Effective for Some Mutations

Added to Collection on: 2021-06-11 16:18:45

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Branches of biology
Nucleic acids
Biology
Genodermatoses
RNA splicing
Spliceosome
Rare diseases
Collagens
Exon skipping
Epidermolysis bullosa
Junctional epidermolysis bullosa
Exon

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'Frameshifting' Therapy for Mast Cell Cancers Reduces Size, Spread

Added to Collection on: 2021-08-10 16:18:45

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Branches of biology
Biology
RNA
Gene expression
Molecular biology
Spliceosome
Mastocytosis
Messenger RNA
Frameshift mutation
Mast cell
Kit
Exon skipping

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Nippon Shinyaku and Capricor Therapeutics Enter into an Exclusive Partnership for ...

Added to Collection on: 2022-01-25 20:10:12

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Life Sciences
Branches of biology
Health sciences
Muscular dystrophy
Food and Drug Administration
Pharmaceuticals policy
Breakthrough therapy
Pharmacy
Regenerative Medicine Advanced Therapy
Exon skipping
Duchenne muscular dystrophy
Exosome
CAP-1002
United States

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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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