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Spinal muscular atrophy

Genetown
Yet-To-Be-Approved Gene Therapy Dubbed More Cost Effective Than Spinraza, Nonprofit Says
Using a calculation, ICER believes the Novartis gene therapy for Spinal Muscular Atrophy will be more cost effective than Biogen’s Spinraza.
December 21, 2018
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2 min read
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Alex Keown
Policy
Novartis’ SMA Gene Therapy Is on the Fast Track for Approval
Novartis’ gene therapy treatment for spinal muscular atrophy could see approval as early as May of this year after the U.S. Food and Drug Administration granted AVXS-101, now dubbed Zolgensma, priority review.
December 3, 2018
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2 min read
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Alex Keown
globalmoments via Getty Images
Drug Development
PTC Therapeutics’ Drug for Spinal Muscular Atrophy Shows Promise in 2 Trials
PTC Therapeutics announced interim data from Part 1 of its open-label clinical trials, FIREFISH and SUNFISH, of risdiplam for Type 1, 2 and 3 spinal muscular atrophy in collaboration with Genentech.
October 3, 2018
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2 min read
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Mark Terry
Business
PTC Therapeutics Presented Updated Interim Data from SMA Clinical Trial
PTC Therapeutics presented updated interim clinical data from Part 1 of its FIREFISH clinical trial of risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy.
June 18, 2018
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3 min read
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Mark Terry
Business
Cytokinetics’ Spinal Muscular Atrophy Drug Shows Promise in Phase II
Cytokinetics released data from its Phase II clinical trial of reldesemtiv in patients with spinal muscular atrophy (SMA).
June 18, 2018
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2 min read
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Mark Terry
Drug Development
Biogen’s First Quarter Driven by MS Drugs and Spinraza
Biogen saw a strong first quarter with 11 percent growth in revenues to $3.1 billion, which were spurred by gains in spinal muscular atrophy (SMA) treatment Spinraza and multiple sclerosis treatment Tecfidera.
April 24, 2018
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3 min read
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Alex Keown
FDA
PTC Therapeutics CEO to Fight for DMD Therapy, Pushes Forward with SMA Treatment
The company believes the Translarna data is compelling and pointed to the success the drug is having in Europe.
January 10, 2018
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3 min read
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Alex Keown
What You Missed: Nippon Fine Chemical Formed Human Barricade to Stop FDA Employee
FDA
FDA Approves First-Ever Treatment for Spinal Muscular Atrophy; Ionis Pharma, Biogen Seal Victory
December 27, 2016
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3 min read
Genetown
This Toddler With A Rare Disease Got A Life-Changing Treatment. Why Can’t All Kids?
December 16, 2016
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1 min read
Astellas Pharma, Proteostasis Therapeutics Forge $1.2 Billion Genetic Disease Drug Development Pact
Drug Development
Watch Out, Ionis Pharma: Could This Little Biotech’s Experimental SMA Treatment Be a Threat?
October 14, 2016
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2 min read
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PRESS RELEASES
Press Releases
FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)
February 13, 2025
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7 min read
Press Releases
FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)
February 13, 2025
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9 min read
Press Releases
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
January 23, 2025
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7 min read
Press Releases
Majority of Children With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
October 14, 2024
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10 min read
Press Releases
Majority of children with spinal muscular atrophy (SMA) treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate
October 14, 2024
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8 min read
Press Releases
Spinal Muscular Atrophy Market Size to Reach US$ 13.0 Billion by 2034, Impelled by Increased Popularity of Gene Therapy
July 17, 2024
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12 min read
Biotech Bay
Five-year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of Spinal Muscular Atrophy (SMA) Achieved or Maintained the Ability to Sit, Stand or Walk
June 7, 2024
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9 min read
Policy
Majority of Newborn Babies With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Able to Sit Independently After 1 Year of Treatment
October 4, 2023
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9 min read
Pharm Country
Biohaven’s Taldefgrobep Alfa Receives EU Orphan Drug Designation for Spinal Muscular Atrophy
July 31, 2023
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6 min read
Biotech Bay
Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk
June 30, 2023
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9 min read
FDA
Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
February 21, 2023
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5 min read
FDA
PerkinElmer Announces its EONIS SCID-SMA Kit is First to Receive Marketing Authorization by U.S. FDA for SMA Screening in Newborns
November 14, 2022
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3 min read
Spinal Muscular Atrophy Treatment Market Size to Hit USD 18.9 BN by 2030
November 7, 2022
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9 min read
Biotech Bay
Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
October 12, 2022
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10 min read
Drug Development
Biophytis Presents Preclinical Efficacy Data for Sarconeos (BIO101) in Spinal Muscular Atrophy (SMA), a Rare Neuromuscular Disease
June 16, 2022
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4 min read
Genetown
New Data Presented at Cure SMA Reveal Residual Unmet Needs in Young SMA Patients Treated With Gene Therapy and Suggest Further Potential of Using SPINRAZA® (nusinersen)
June 15, 2022
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10 min read
Policy
FDA Approves Genentech’s Evrysdi (risdiplam) for Use in Babies Under Two Months With Spinal Muscular Atrophy (SMA)
May 31, 2022
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9 min read
Policy
FDA Approves Label Extension for Evrysdi® for Infants with Spinal Muscular Atrophy Under 2 Months Old
May 31, 2022
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8 min read
Biotech Bay
New Data for Genentech’s Evrysdi (risdiplam) Demonstrate Long-Term Efficacy and Safety in a Broad Population of People With Spinal Muscular Atrophy (SMA)
March 16, 2022
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10 min read
Policy
FDA Grants Evrysdi® Priority Review Based on Results From Treating Pre-Symptomatic Infants with Spinal Muscular Atrophy
January 25, 2022
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8 min read
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