Tag: Rare diseases

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Recent Related Articles to Rare diseases

Amylyx Pharmaceuticals Discontinues Orion Program of Ax0035

Added to Collection on: 2025-08-28 17:07:49

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Rare diseases
Sodium phenylbutyrate/ursodoxicoltaurine
Cognitive disorders
Amylyx Pharmaceuticals
Corticobasal syndrome
Progressive supranuclear palsy
Ursodoxicoltaurine
Congenital hyperinsulinism
Hyperinsulinemic hypoglycemia
PlayStation Portable
Hyperinsulinism
PSP

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Biohaven's rare disease drug is 'ready to ship on Day 1' following FDA approval

Added to Collection on: 2025-09-04 17:25:09

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Rare diseases
Autosomal dominant disorders
Spinocerebellar ataxia
Rimegepant
Migraine
AbbVie
Cerebellar ataxia
Ataxia
Pfizer

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Avidity Biosciences Completes $690 Million Share Offering - AInvest

Added to Collection on: 2025-09-15 20:24:34

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Muscular dystrophy
Rare diseases
Nucleic acids
Dystrophy
Medical terminology
DM1
Duchenne muscular dystrophy
Oligonucleotide
Facioscapulohumeral muscular dystrophy
Biotechnology
Duchenne
Avidity Biosciences Inc.

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Ultragenyx's Glycogen Storage Disease Gene Therapy DTX401 Continues to Enable ... - CGTLive®

Added to Collection on: 2025-09-16 21:38:24

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Hepatology
Inborn errors of carbohydrate metabolism
Rare diseases
Ultragenyx
Glycogen storage disease type I
Glycogen storage disease
Gene therapy
Blood sugar level

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Arvinas reboots, cuts more staff; Porges joins Lazard - BioPharma Dive

Added to Collection on: 2025-09-19 15:30:26

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Rare diseases
GLP-1 receptor agonists
PEPTIDE THERAPEUTICS
Garetosmab
Vertex Pharmaceuticals
Thalassemia
Regeneron Pharmaceuticals
Proteolysis targeting chimera
Sickle-cell disease
Novo Nordisk
Pfizer
Fibrodysplasia ossificans progressiva
United States

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Lack of CFAP54 causes primary ciliary dyskinesia in a mouse model and human patients

Added to Collection on: 2025-09-28 05:33:20

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Rare diseases
Ciliopathy
Infertility
Primary ciliary dyskinesia
PCD
Frameshift mutation
Exome
Cilium

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Health Canada approves Santhera's Agamree for DMD treatment

Added to Collection on: 2025-10-06 07:39:34

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Muscular dystrophy
Rare diseases
Duchenne muscular dystrophy
Vamorolone
Catalyst Pharmaceuticals
DMD
Priority review
Dystrophin
Prosensa

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$AISP stock is up 16% today. Here's what we see in our data.

Added to Collection on: 2025-10-06 15:13:12

Tags for this article:

Rare diseases
Myotonia
Myotonic dystrophy
Muscular dystrophy
Dystrophy
Duchenne muscular dystrophy
DM1
Myotonin-protein kinase
Glycogen storage disease type II

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Immix Biopharma, Inc. to Present NXC-201 Abstract at ASH 67th Annual Meeting

Added to Collection on: 2025-10-06 20:46:43

Tags for this article:

Rare diseases
Myotonia
Myotonic dystrophy
Muscular dystrophy
Dystrophy
Duchenne muscular dystrophy
DM1
Myotonin-protein kinase
Glycogen storage disease type II

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Gene Behind Child Brain Disorder Also Tied to Parkinson's Disease - Neuroscience News

Added to Collection on: 2025-10-09 20:15:56

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Neurological disorders
Rare diseases
Syndromes
Clinical neuroscience
Cognitive disorders
Vici syndrome
Neurodegenerative disease
Neurodevelopmental disorder
Autophagy
Dementia
Zhenyu Yue

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