Tag: Rare diseases

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Recent Related Articles to Rare diseases

Johns Hopkins Medicine-Led Team Develops Fluid Biomarker for Early Detection of ...

Added to Collection on: 2024-03-18 18:59:44

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Cognitive disorders
Rare diseases
Dementia
Frontal lobe
Frontotemporal dementia
Temporal lobe
ALS
TAR DNA-binding protein 43
C9orf72
Genetics of amyotrophic lateral sclerosis

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Johns Hopkins Medicine-Led Team Develops Fluid Biomarker for Early Detection of ALS and FTD

Added to Collection on: 2024-03-18 17:23:11

Tags for this article:

Cognitive disorders
Rare diseases
Dementia
Frontal lobe
Frontotemporal dementia
Temporal lobe
ALS
TAR DNA-binding protein 43
C9orf72
Genetics of amyotrophic lateral sclerosis

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Johns Hopkins Leads Development of ALS, FTD Early-Detection Biomarker | Mirage News

Added to Collection on: 2024-03-18 17:06:16

Tags for this article:

Cognitive disorders
Rare diseases
Dementia
Frontal lobe
Frontotemporal dementia
Temporal lobe
ALS
TAR DNA-binding protein 43
C9orf72
Genetics of amyotrophic lateral sclerosis

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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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The prevalence and clinical features of MYO7A-related hearing loss including DFNA11 ...

Added to Collection on: 2024-04-09 16:56:07

Tags for this article:

Cytoskeleton
MYO7A
Proteins
Usher syndrome
Rare diseases
Channelopathies
ARNSHL

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Long-Term Data Affirm Efficacy of Eculizumab in Refractory Generalized MG

Added to Collection on: 2024-04-15 20:56:51

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Monoclonal antibodies
Orphan drugs
Immunosuppressants
Autoimmune diseases
Eculizumab
Myasthenia gravis
Rare diseases
Ravulizumab
Apheresis
Atypical hemolytic uremic syndrome

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Safety Considerations for Ravulizumab in NMOSD: Sean Pittock, MD - NeurologyLive

Added to Collection on: 2024-04-23 19:08:11

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Monoclonal antibodies
Orphan drugs
Autoimmune diseases
Rare diseases
Neuromyelitis optica spectrum disorder
Satralizumab
Inebilizumab
Eculizumab
Multiple sclerosis
Aquaporin-4
Ravulizumab
Therapy

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Non-Cystic Fibrosis Bronchiectasis Pipeline Drugs Analysis Report, 2024 Updates: FDA ...

Added to Collection on: 2024-04-24 15:11:57

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Rare diseases
Respiratory diseases
Bronchiectasis
Cystic fibrosis
Boehringer Ingelheim
Pseudomonas aeruginosa
Non-Cystic Fibrosis Bronchiectasis
DelveInsight | Insmed Inc

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Groundbreaking Trial Targets Genetic Cause of Epilepsy - Checkup Newsroom

Added to Collection on: 2024-05-02 21:34:22

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Epilepsy
Dravet syndrome
SCN1A
Rare diseases
Biotechnology
Genetic engineering
Epilepsy-intellectual disability in females
Dravet Syndrome Foundation
M. Scott Perry

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Remote pulmonary function testing allows for early identification of need for non-invasive ...

Added to Collection on: 2024-05-02 11:07:14

Tags for this article:

Respiratory diseases
Rare diseases
Respiratory therapy
Non-invasive ventilation
ALS
Pulmonary function testing
Respiratory failure
Telehealth

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