News
Drug Development
FDA
Manufacturing
Deals
Business
Job Trends
Cell and Gene Therapy
Cancer
Opinions
Press Releases
All News & Releases
Podcasts
Events
Jobs
Companies
Hotbeds
More
Reports
Career Advice
NextGen: Top Startups to Watch
40 Under 40
Best Places to Work
Employer Resources
Advertise
Advertise
Talent Solutions
Post Jobs
Submit a Press Release
Submit an Event
SUBSCRIBE
Menu
SUBSCRIBE
Show Search
News
Drug Development
FDA
Manufacturing
Deals
Business
Job Trends
Cell and Gene Therapy
Cancer
Opinions
Press Releases
All News & Releases
Podcasts
Events
Jobs
Companies
Hotbeds
More
Reports
Career Advice
NextGen: Top Startups to Watch
40 Under 40
Best Places to Work
Employer Resources
Advertise
Advertise
Talent Solutions
Post Jobs
Submit a Press Release
Submit an Event
Search Query
Submit Search
Spinal muscular atrophy
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
·
3 min read
·
Alex Keown
FDA
FDA Approves First-Ever Treatment for Spinal Muscular Atrophy; Ionis Pharma, Biogen Seal Victory
December 27, 2016
·
3 min read
Genetown
This Toddler With A Rare Disease Got A Life-Changing Treatment. Why Can’t All Kids?
December 16, 2016
·
1 min read
Drug Development
Watch Out, Ionis Pharma: Could This Little Biotech’s Experimental SMA Treatment Be a Threat?
October 14, 2016
·
2 min read
World’s First Child-Exoskeleton For Spinal Muscular Atrophy, CSIC/Politechnic Univerity of Madrid Reveals
June 9, 2016
·
3 min read
Business
Pfizer Abandons $70 Million SMA Deal With Repligen Corporation
January 30, 2015
·
2 min read
BioMidwest
Candidate Drug Provides Benefit in Spinal Muscular Atrophy Animal Models, Human Molecular Genetics Reveals
June 5, 2013
·
1 min read
Genetown
Link Found Between ALS and Spinal Muscular Atrophy, Harvard Medical School Study
September 28, 2012
·
1 min read
Drug Development
Spinal Muscular Atrophy May Also Affect the Heart, Nationwide Children’s Hospital Study Finds
August 13, 2010
·
1 min read
BioMidwest
Study Finds Screening For Spinal Muscular Atrophy Not Cost Effective, Society for Maternal-Fetal Medicine
February 5, 2010
·
1 min read
PRESS RELEASES
Press Releases
Dubai’s Medcare Hospital Becomes the World’s First to Treat an Adult Spinal Muscular Atrophy (SMA) Patient Outside the US
April 28, 2026
·
2 min read
Press Releases
Novartis receives positive CHMP opinion for Itvisma® for spinal muscular atrophy (SMA)
April 24, 2026
·
8 min read
Press Releases
Scholar Rock Resubmits Biologics License Application (BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)
March 31, 2026
·
9 min read
Press Releases
FDA Approves High Dose Regimen of SPINRAZA® for Spinal Muscular Atrophy, Reflecting Progress Made Possible Through Decades of MDA Supported Research
March 30, 2026
·
4 min read
Press Releases
FDA Approves New High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy
March 30, 2026
·
13 min read
Press Releases
Biogen Presents Additional Salanersen Data Showing New Motor Milestones Achieved in Children with SMA Previously Treated with Gene Therapy
March 11, 2026
·
12 min read
Press Releases
Voyager Reports Fourth Quarter and Full Year 2025 Financial and Operating Results
March 9, 2026
·
16 min read
Press Releases
GEMMABio Announces First Patient Dosed in CHARISMA Trial of GB221, Investigational Next‑Generation Gene Therapy for Spinal Muscular Atrophy Type 1 (SMA1)
February 26, 2026
·
5 min read
Press Releases
Nature Medicine Publishes Results from the Pivotal DEVOTE Study of High-Dose Regimen of Nusinersen in Spinal Muscular Atrophy
February 4, 2026
·
10 min read
Press Releases
NMD Pharma announces topline results from its Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2
February 3, 2026
·
10 min read
Press Releases
Biogen Receives European Commission Approval for High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy
January 13, 2026
·
13 min read
Press Releases
Orsini Selected as Specialty Pharmacy Partner for ITVISMA® (onasemnogene abeparvovec-brve)
December 3, 2025
·
2 min read
Press Releases
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)
November 25, 2025
·
8 min read
Press Releases
High Dose Regimen of Nusinersen Receives Positive CHMP Opinion for the Treatment of Spinal Muscular Atrophy
November 17, 2025
·
13 min read
Press Releases
FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)
February 13, 2025
·
7 min read
Press Releases
FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)
February 13, 2025
·
9 min read
Press Releases
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
January 23, 2025
·
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
·
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
·
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
·
12 min read
