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 - Tuesday, May 12, 2026
See today's BioWorld
Home » Questions outnumber answers for rare disease community at NIH gene therapy workshop
To read the full story, subscribe or sign in.

Questions outnumber answers for rare disease community at NIH gene therapy workshop

Aug. 22, 2018
By Michael Fitzhugh
An NIH workshop aimed at promoting the efficient development and regulation of gene therapies highlighted a crucial bottleneck for the field Tuesday, as policy members of the rare disease community raised substantial questions about how to value and pay for the therapies. With three gene therapies already approved in the U.S. and a growing queue of new ones in development, panelists painted a picture of financial and institutional innovation struggling to keep pace with medical innovation.
BioWorld

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for May 12, 2026.
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for May 12, 2026
  • Brain maze

    Alzheimer’s, beyond the brain

    BioWorld
    Researchers at Daping Hospital in China have reported that liver-targeted delivery of the APOE3-Christchurch (APOE3Ch) variant, a rare protective form of...
  • Concept art for "unlocking the secrets of the mind"

    New assay for drug discovery against chemo-induced peripheral neuropathy

    BioWorld Science
    A group led by researchers at Boston Children’s Hospital established a scalable and reproducible model of paclitaxel-induced axon degeneration and neurotoxicity...
  • Hantavirus zoonotic spillover illustration

    First cruise ship hantavirus genome points to zoonotic spillover

    BioWorld
    The initial appraisal of the first complete genome sequence of a hantavirus isolated from a patient in Switzerland who was a passenger on the cruise ship MV...
  • 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