Fierce Pharma
Fierce Biotech
Fierce Healthcare
Fierce Life Sciences Events
Advertise
About Us
Biotech
Cell & Gene Therapy
Clinical Data
Venture Capital
Deals
Research
Medtech
Devices
Diagnostics
AI and Machine Learning
CRO
Special Reports
Trending Topics
Biologics
Clinical Development
Fierce 50
Special Report
Awards Gala
Resources
Fierce Events
Industry Events
Webinars
Podcasts
Whitepapers
Survey
Events
Subscribe
Trending T
Biotech
Cell & Gene Therapy
Clinical Data
Venture Capital
Deals
Research
Medtech
Devices
Diagnostics
AI and Machine Learning
CRO
Special Reports
Trending Topics
Biologics
Clinical Development
Fierce 50
Special Report
Awards Gala
Resources
Fierce Events
Industry Events
Webinars
Podcasts
Whitepapers
Survey
Events
Subscribe
Fierce Pharma
Fierce Biotech
Fierce Healthcare
Fierce Life Sciences Events
Advertise
About Us
lysosomal storage disorders
Biotech
Denali rare disease drug clears safety study, reduces biomarkers
Denali's drug met its primary safety endpoints and reduced key biomarkers of Hunter syndrome in a phase 1/2 trial, the biotech announced.
Darren Incorvaia
Feb 6, 2025 12:05pm
'Clinical intuition' led FDA advisors to back Zevra's rare disease med
Aug 5, 2024 10:35am
Takeda eyes cures for billion-dollar gene therapy spending spree
May 4, 2022 4:00am
Avrobio gene therapy shows signs of durability in rare disorder
Feb 9, 2022 10:46am
CRISPR corrects genetic disease before and after birth in mice
Jul 13, 2021 5:00am
Chiesi creates Boston rare disease unit ahead of Fabry launch
Feb 7, 2020 8:45am
