Tag: Rare diseases

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Recent Related Articles to Rare diseases

BioMarin Presents Real-World Evidence Further Supporting Safety and Efficacy of ... - Quantisnow

Added to Collection on: 2024-11-16 22:06:53

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Growth disorders
Dwarfism
Rare diseases
Congenital disorders
Skeletal system
Achondroplasia
Vosoritide
BioMarin Pharmaceutical
Fibroblast growth factor receptor 3
Hypochondroplasia
Short stature
Epiphyseal plate
BioMarin Pharmaceutical Inc.
Voxzogo
Noonan
Turner

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Citizen Health secures $14.5M in seed funding for rare disease drug development

Added to Collection on: 2024-11-19 20:10:25

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Medical terminology
National Institutes of Health
Rare diseases
Rare disease
Rare Diseases Clinical Research Network
Patient advocacy
Disease
Medical diagnosis
Rare Diseases Clinical Research Network Contact Registry
Genetic Alliance UK
Citizen Health

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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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Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments

Added to Collection on: 2024-11-19 20:05:10

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Rare diseases
Danon disease
Gene delivery
Medical genetics
Molecular biology
LAMP2
Gene therapy
Leerink Partners
Danon
A501
Rocket Pharmaceuticals Inc

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1st gene therapy delivered directly into brain approved - Becker's Hospital Review

Added to Collection on: 2024-11-20 15:39:38

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Biotechnology
Rare diseases
PTC Therapeutics
South Plainfield, New Jersey
Neurosurgery
Aromatic L-amino acid decarboxylase deficiency
Gene therapy
Stereotactic surgery
Cannula

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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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Raising Awareness of Pulmonary Hypertension: What You Need to Know

Added to Collection on: 2024-11-26 22:24:23

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Hypertension
Rare diseases
Heart diseases
Medical emergencies
Pulmonary hypertension
Lung disorders
Heart failure
Respiratory disease
Pulmonary embolism
Cardiology
Pulmonary arterial hypertension
Chronic thromboembolic pulmonary hypertension

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Neurohormonal activation pattern in patients with atrial septal defect | Scientific Reports

Added to Collection on: 2024-12-02 18:30:53

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Peptide hormones
Hypertension
Endocrinology
Rare diseases
Renal physiology
Pulmonary hypertension
Angiotensin
Natriuretic peptide
Renin
Aldosterone
Endothelin
Pulmonary arterial hypertension

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Rilzabrutinib Shines in Phase 3 Trial of Tough-to-Treat Immune Thrombocytopenia

Added to Collection on: 2024-12-08 15:37:37

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Transfusion medicine
Rare diseases
Coagulation system
Immune thrombocytopenic purpura
Orphan drugs
Thrombocytopenia
Platelet
Placebo
Coagulation
Thrombocytopenic purpura
Fostamatinib
Plateletpheresis
David J. Kuter

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