Tag: Risdiplam

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Recent Related Articles to Risdiplam

Roche gets FDA priority review for Evrysdi to treat SMA in new-borns

Added to Collection on: 2022-01-27 11:17:19

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Spinal muscular atrophy
Neurological disorders
Drugs
Orphan drugs
Health sciences
Risdiplam
PTC Therapeutics
SMA
New Drug Application
SMA Foundation

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Roche Releases More Evrysdi Data For Very Young Infants With SMA - Benzinga

Added to Collection on: 2022-04-30 07:09:03

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Spinal muscular atrophy
Neurological disorders
Branches of biology
Neurodegenerative disorders
SMN1
Risdiplam
SMN2
SMA
Muscle atrophy
Branaplam
Nusinersen
Roche Holding AG's
Roche Holding AG

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No New Issues for Evrysdi Reported in Compassionate Use Program - SMA News Today

Added to Collection on: 2022-07-22 15:32:36

Tags for this article:

Neurological disorders
Branches of biology
Neurodegenerative disorders
Spinal muscular atrophy
Orphan drugs
Neurogenetic disorders
Motor neuron diseases
Risdiplam
SMA
Muscle atrophy
PTC Therapeutics
Germany

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Roche Evrysdi improves/maintains motor function in SMA patients in new 2-year data

Added to Collection on: 2022-10-12 09:05:42

Tags for this article:

Neurological disorders
Spinal muscular atrophy
Neurodegenerative disorders
Nusinersen
Onasemnogene abeparvovec
Survival of motor neuron
Risdiplam
SMA
Epigenetics of neurodegenerative diseases
SMA Foundation
Microbe Roche

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Roche presents positive results for spinal muscular atrophy treatment Evrysdi in babies

Added to Collection on: 2023-10-06 17:45:15

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Spinal muscular atrophy
Orphan drugs
Risdiplam
SMA
SMN2
Myostatin

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diversity of splicing modifiers acting on A-1 bulged 5 - Oxford Academic

Added to Collection on: 2024-03-30 15:26:11

Tags for this article:

Spliceosome
RNA splicing
Gene expression
RNA
Alternative splicing
Small nuclear RNA
SnRNP
U1 spliceosomal RNA
LSm
Intron
Spinal muscular atrophy
Risdiplam
SMN

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7MM Spinal Muscular Atrophy Opportunity Assessment and - GlobeNewswire

Added to Collection on: 2024-07-29 14:30:42

Tags for this article:

Spinal muscular atrophy
Orphan drugs
Motor neuron diseases
Biotechnology
Nusinersen
Onasemnogene abeparvovec
Gene therapy
SMA
Risdiplam
Biogen
SMN2
ResearchAndMarkets.com

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Crystal Proud, MD, on Improving Newborn Screening for Spinal Muscular Atrophy

Added to Collection on: 2024-09-03 17:42:03

Tags for this article:

Spinal muscular atrophy
Orphan drugs
Applied genetics
Nusinersen
Onasemnogene abeparvovec
Gene therapy
Biogen
Risdiplam
Adeno-associated virus

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What Do SMA Caregivers Want? - Managed Healthcare Executive

Added to Collection on: 2024-11-05 16:24:37

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Spinal muscular atrophy
Orphan drugs
Onasemnogene abeparvovec
Caregiver
Risdiplam
Nusinersen
Novartis

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Gene Therapy Improves Motor Function in Spinal Muscular Atrophy Type 2 - eMPR.com

Added to Collection on: 2024-12-30 20:13:53

Tags for this article:

Spinal muscular atrophy
Orphan drugs
Risdiplam
Onasemnogene abeparvovec
Nusinersen
Gene therapy
ClinicalTrials.gov

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