Tag: Duchenne muscular dystrophy

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Recent Related Articles to Duchenne muscular dystrophy

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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Avatar's Motion AI Tech Helps Researchers Detect Rare FA and DMD Disorders Six Months ...

Added to Collection on: 2023-01-22 20:19:23

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Muscular dystrophy
Neurological disorders
Organ systems
RTT
Branches of biology
Duchenne muscular dystrophy
Dystrophin
Friedreich's ataxia
Ataxia
Nikolaus Friedreich

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Family raises awareness on the affects of Duchenne Muscular Dystrophy | News - KHQ.com

Added to Collection on: 2023-09-21 19:54:49

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Muscular dystrophy
Duchenne muscular dystrophy
Dystrophin
Dystrophy
DMD
Dystrophinopathy
Becker muscular dystrophy

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Gene therapy death was caused by an unknown risk of the virus used, study suggests

Added to Collection on: 2023-09-28 16:02:43

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Biotechnology
Applied genetics
Gene delivery
Medical genetics
Molecular genetics
Gene therapy
Cure Rare Disease
Adeno-associated virus
Duchenne muscular dystrophy
Jesse Gelsinger
Pfizer-BioNTech COVID-19 vaccine
Oncolytic AAV

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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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[Ad hoc announcement pursuant to Art. 53 LR] Roche - GlobeNewswire

Added to Collection on: 2023-10-30 20:09:32

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Muscular dystrophy
Biotechnology
Genetic engineering
Breakthrough therapy
Delandistrogene moxeparvovec
Gene therapy
Duchenne muscular dystrophy
Sarepta Therapeutics
Dystrophin
DMD
Dystrophin-associated protein complex
United States
Roche Group
Duchenne
Basel
Roche Founded

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CHMP Issues Negative Opinion for Renewal of Conditionally Approved Therapy Ataluren

Added to Collection on: 2024-01-31 20:12:20

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Clinical research
Orphan drugs
Ataluren
Cystic fibrosis
Oxadiazoles
Duchenne muscular dystrophy
PTC Therapeutics
Nonsense mutation
Placebo

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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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Patient dies in Pfizer study of Duchenne gene therapy | BioPharma Dive

Added to Collection on: 2024-05-07 21:32:57

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Clinical research
Design of experiments
Muscular dystrophy
Genetic engineering
Clinical trials
Pfizer
Duchenne muscular dystrophy
Sarepta Therapeutics
Gene therapy
Placebo-controlled study
Domagrozumab

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Cell and Gene Therapies Still Have Long Way to Go in Fulfilling Promise | BioSpace

Added to Collection on: 2024-05-10 14:11:49

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Biotechnology
Genetic engineering
Molecular biology
American Society of Gene and Cell Therapy
Medical genetics
Gene delivery
Gene therapy
Biopharmaceutical
Duchenne muscular dystrophy
CRISPR
Pfizer
Vertex Pharmaceuticals

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