Tag: Rare diseases

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Recent Related Articles to Rare diseases

Updated Data Suggests Sangamo's Gene Therapy ST-920 Improves Renal Function in ...

Added to Collection on: 2025-02-06 23:37:08

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Lysosomal storage diseases
Rare diseases
Sangamo Therapeutics
Fabry disease
Gene therapy
Epidermal growth factor receptor

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aHUS | Alexion US Site

Added to Collection on: 2025-02-07 15:13:13

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Syndromes
Blood disorders
Rare diseases
Autoimmune diseases
Atypical hemolytic uremic syndrome
Disease
Immune system
Sjögren's disease
Hemolytic–uremic syndrome
Hemolysis

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Mechanism For Treating Muscle Wasting Discovered | Mirage News

Added to Collection on: 2025-02-12 15:52:28

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Muscular dystrophy
Cytoskeleton
Rare diseases
Utrophin
Duchenne muscular dystrophy
Dystrophin
Dystrophin-associated protein complex
Skeletal muscle
Gene therapy
Sarcospan

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New Functional MRI Model Shows High Accuracy in Differentiating Between MS and NMOSD

Added to Collection on: 2025-02-16 20:04:41

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Multiple sclerosis
Autoimmune diseases
Rare diseases
Neuromyelitis optica spectrum disorder
MOG antibody disease
Functional magnetic resonance imaging
Imaging biomarker
Fatigue
Myelin oligodendrocyte glycoprotein
Magnetic resonance imaging
Research in multiple sclerosis

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Clinical trial provides hope for child with rare genetic disorder - WCVB-TV

Added to Collection on: 2025-03-01 13:35:33

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Rare diseases
Lipid storage disorders
Leukodystrophies
Canavan disease
Magnetic resonance imaging
Therapy
Noa

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Blueprint Medicines Highlights Leading Portfolio of Mast Cell-Ta - GuruFocus

Added to Collection on: 2025-03-01 12:45:26

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Orphan drugs
Rare diseases
Immune system disorders
Mastocytosis
Avapritinib
Mast cell leukemia
Medication
Midostaurin

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Extracellular Vesicles Released by Genetically Modified Macrophages to Treat Parkinson's ...

Added to Collection on: 2025-03-04 15:26:40

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Lipid storage disorders
Rare diseases
Senescence
Neurodegenerative disorders
Batten disease
Neurodegenerative disease
Neuronal ceroid lipofuscinosis
Infantile neuronal ceroid lipofuscinosis
Degenerative disease
NCL
Jansky–Bielschowsky disease
Nazira El-Hage

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Roche Holding AG to Present New Data At the Muscular Dystrophy Association Conference ...

Added to Collection on: 2025-03-17 14:11:07

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Muscular dystrophy
Rare diseases
Biotechnology
Delandistrogene moxeparvovec
Duchenne muscular dystrophy
Spinal muscular atrophy
Muscular Dystrophy Association
Dystrophy
Roche Holding AG

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CGTLive®'s Weekly Rewind – March 21, 2025

Added to Collection on: 2025-03-21 19:08:22

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Biotechnology
Applied genetics
Muscular dystrophy
Rare diseases
Bioethics
Gene therapy
Duchenne muscular dystrophy
Delandistrogene moxeparvovec
Familial amyloid polyneuropathy
Therapy
Sarepta Therapeutics
Toshifumi Yokota

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Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD

Added to Collection on: 2025-03-21 18:00:01

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Biotechnology
Muscular dystrophy
Applied genetics
Delandistrogene moxeparvovec
Sarepta Therapeutics
Rare diseases
Duchenne muscular dystrophy
Gene therapy
Muscular Dystrophy Association
Dystrophin
Adeno-associated virus
Dystrophin-associated protein complex
DALLAS

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