Tag: Muscular dystrophy

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Recent Related Articles to Muscular dystrophy

Goldman Sachs Bullish On Avidity Biosciences, Cites Scalable RNA Therapeutics Platform ...

Added to Collection on: 2024-09-24 18:38:10

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Muscular dystrophy
Rare diseases
Duchenne muscular dystrophy
Facioscapulohumeral muscular dystrophy
Dystrophy
Duchenne
Dystrophin

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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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Boy with Duchenne's Gets Gene Therapy 3 Days Before He's Ineligible (Exclusive)

Added to Collection on: 2024-10-05 14:21:00

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Muscular dystrophy
Duchenne muscular dystrophy
Muscular Dystrophy Association
Delandistrogene moxeparvovec
Dystrophin
Dystrophy
Biostrophin
Limb–girdle muscular dystrophy
Hudson Sanford
Melanie Sanford Sanford
Melanie Sanford

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AAV Vectors in Gene Therapy: Overcoming Barriers for Muscular Dystrophy Treatment

Added to Collection on: 2024-10-28 19:07:18

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Muscular dystrophy
Applied genetics
Gene delivery
Medical genetics
Gene therapy
Adeno-associated virus
Limb–girdle muscular dystrophy
Duchenne muscular dystrophy
Dystrophin
Skeletal muscle
Gene therapy of the human retina
Oncolytic AAV

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FDA grants ODD status to MDL-101 for LAMA2-CMD treatment

Added to Collection on: 2024-10-28 14:58:38

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Applied genetics
Muscular dystrophy
Gene delivery
Medical genetics
Molecular biology
Laminin subunit alpha-2
Congenital muscular dystrophy
Gene therapy
Adeno-associated virus
LAMA2 related congenital muscular dystrophy

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Sarepta Therapeutics Announces Third Quarter 2024 Financial Results and Recent ...

Added to Collection on: 2024-11-06 22:03:35

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Biotechnology
Corvallis, Oregon
Sarepta Therapeutics
Delandistrogene moxeparvovec
Duchenne muscular dystrophy
Gene therapy
Muscular dystrophy
Duchenne
Magnetic resonance imaging
Sarepta Therapeutics Inc.

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Regenxbio eyes 2026 filing for Duchenne muscular dystrophy gene therapy

Added to Collection on: 2024-11-19 17:37:27

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Muscular dystrophy
Biotechnology
Rare diseases
Corvallis, Oregon
Sarepta Therapeutics
Duchenne muscular dystrophy
Delandistrogene moxeparvovec
Gene therapy
Dystrophin
REGENXBIO

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Genethon's Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With ...

Added to Collection on: 2024-11-25 22:12:03

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Applied genetics
Biotechnology
Muscular dystrophy
Rare diseases
Medical genetics
Gene therapy
Duchenne muscular dystrophy
Crigler–Najjar syndrome
Adeno-associated virus
Follistatin
Steve Wilton
Genethon

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Real-World Challenges in DMD PMO Treatment - American Journal of Managed Care

Added to Collection on: 2024-11-25 18:06:45

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Muscular dystrophy
Morpholines
Therapeutic gene modulation
Antisense RNA
Rare diseases
Sarepta Therapeutics
Duchenne muscular dystrophy
Casimersen
Eteplirsen
Morpholino
Dystrophin
Adherence
Duchenne

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New drug shows promise against Duchenne muscular dystrophy - McGill University

Added to Collection on: 2024-12-16 14:12:52

Tags for this article:

Muscular dystrophy
Duchenne muscular dystrophy
Dystrophin
Stem cell
Gene therapy
Skeletal muscle
Becker muscular dystrophy
Mdx mouse

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