BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld Science
  • BioWorld Asia
  • Data Snapshots
    • Biopharma
    • Medical technology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Med-tech outlook 2026
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • Bioworld 2025 review
    • BioWorld MedTech 2025 review
    • BioWorld Science 2025 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Thursday, April 2, 2026
See today's BioWorld
Home » FDA ‘blacklist’ in DMD? Legislator slams PTC turndown
To read the full article sign up for free or sign in.

FDA ‘blacklist’ in DMD? Legislator slams PTC turndown

March 11, 2026
By Randy Osborne
No Comments
The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.
BioWorld Regulatory Musculoskeletal Small molecule U.S. FDA

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for April 1, 2026.
  • Vials, syringes, and pills

    With improved technologies, biomarkers, failed drugs may come into their own

    BioWorld
    At BioEurope Spring 2026, pharma representatives and investors shared their thoughts about current and future landscapes of different disease areas, and on how to...
  • Comparison of neurons in a healthy brain and nerve cells in neurodegenerative disease with amyloid plaques

    Small-molecule TREM2 agonist advances to treat Alzheimer’s

    BioWorld Science
    Microglia play a central role in the neuroinflammation associated with Alzheimer’s disease (AD). These cells act as the brain’s immune system and respond to...
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for March 31, 2026
  • Amyloid beta peptides on neurons

    Amlogenyx’s AM-805 reduces amyloid plaques in AD

    BioWorld Science
    The potent carboxypeptidase enzyme protective protein cathepsin A (PPCA) is known to cleave the C-terminus of amyloid-β42, responsible for aggregation and...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Medical technology
    • Newco news
    • Opinion
    • Regulatory
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/metabolic
    • Immune
    • Infection
    • Neurology/psychiatric
    • NME Digest
    • Patents
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2026. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing