when

Latest
Archive

by source

All Sources
Sources Page

department

Clinical
Regulatory
Medical Affairs
Commercial
R&D / Discovery
Biostatistics / Data
Manufacturing / CMC
Market Access

therapeutic area

Oncology
Immunology
Neuroscience
Cardiovascular
Metabolic
Rare Disease
Infectious Disease

location & type

Remote Only
US Only
California
Massachusetts
Internships
Phase 3 Roles
All Jobs →

Sign In Subscribe

pharmadog

fetch the data · sniff the signal

Discover

Jobs
News

Hubs

Topics
Patent cliff
Publications

Tools

Compare
Search
Bookmarks

Trust

About
Sources
Contact

Legal

Privacy
Terms
Pricing

© 2026 pharmadog.xyz

made by humans and a good dog

home
jobs
news
search

Rare Disease · news · pharmadog

← back to Rare Disease

News mentioning Rare Disease

220 stories

16hUltragenyx, after setbacks, nabs first gene therapy approvalbiopharma dive

17hSTAT+: FDA approves Ultragenyx gene therapystat

18hSTAT+: Pharmalittle: We’re reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and morestat

18hRegeneron wins FDA approval to challenge Ipsen for rare bone disease marketbiospace

19hUltragenyx bags first gene therapy approval as previously rejected Sanfilippo asset awaits decisionbiospace

20hFDA approves Ultragenyx's gene therapy Genglycos for glycogen storage disorderendpoints

23hReduce protein to increase longevity? Nutrition experts say that’s a bad ideastat

23hOpinion: Capricor’s Duchenne muscular dystrophy drug and the countless tiny decisions that can make or break a studystat

1d7 biopharma M&A deals where layoffs followedbiospace

1dWhy pediatric rare oncology outperforms in a volatile biotech marketbiospace

1dRecon: FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation challengesraps

1dSTAT+: Regeneron drug for disease that causes dangerous bone growth earns FDA approvalstat

1dKolon TissueGene cuts 37 US staffers after Phase 3 failbiospace

1dTrump picks White House aide Heidi Overton as FDA chief: Reportsbiospace

1dSTAT+: In wake of boy’s death, gene editing experts dissect Huidagene’s decisionsstat

2dBioMarin acquires experimental drug for rare bone disorder in $275M dealbiopharma dive

2dBioMarin does another small deal to get early-stage bone disease assetendpoints

2dLEO adds to dermatology portfolio with $435M deal for late-stage rare disease drugbiospace

2dAmylyx eyes FDA filing for GLP-1 blocker after late-stage win in post-bariatric hypoglycemiabiospace

3dLeo Pharma to license Tanabe’s rare disease drug for pain with sun exposureendpoints

3dAssessing shared failures with AI is key to cell and gene therapy’s evolutionbiospace

3dAstraZeneca pulls plug on late-stage lung cancer trial, taking another clinical blowbiospace

3dAstraZeneca pulls plug on late-stage lung cancer trial, taking another clinical blowbiospace

4dAsia-Pacific Roundup: Saudi FDA streamlines rare disease drug developmentraps

6dSTAT+: FDA approves Bristol multiple myeloma treatment, marking debut of novel drug classstat

6dPressure piles on Amgen after EU pulls Tavneos, flags 'serious breaches' in pivotal studybiospace

6dAfter 2 undisclosed gene therapy deaths, can China’s alternative regulatory path still be trusted?fiercebiotech

6d‘We’re not afraid of competition’: BioCryst CEO sets out strategy after scrapping internal discoveryfiercebiotech

7dSTAT+: Commure terminates payments under its customer referral programs tied to AI productsstat

7d'Incorrect and misleading' data prompted decision to pull Tavneos in Europeendpoints

← prevpage 1 of 8next →