rare disease

J&J targets rare maternal-fetal diseases: 'We're going for this. No one else has.'
Fierce Biotech
Tue, 04/2/24 - 11:49 am
JNJ
rare disease
maternal-fetal diseases
FNAIT
nipocalimab
AstraZeneca Gets FDA Approval in Boost to Rare Disease Franchise
BioSpace
Mon, 04/1/24 - 11:58 am
AstraZeneca
FDA
rare disease
danicopan
PNH
Amgen details rare disease ambitions following $28B Horizon acquisition
Medical Marketing and Media
Sun, 02/25/24 - 09:04 pm
Amgen
Horizon Therapeutics
rare disease
M&A
Roche, Horizon lead way for pharma reputation among rare disease patient groups
Medical Marketing and Media
Wed, 08/23/23 - 05:49 pm
Roche
Horizon Pharma
reputation
rare disease
Natural History Studies Drive Data Sharing, Drug Approval
BioSpace
Mon, 05/1/23 - 10:07 pm
rare disease
data sharing
natural history database
Ex-Takeda exec hits the ground running at the helm of rare disease biotech — with 3 priorities off the bat
Endpoints
Fri, 05/20/22 - 10:34 am
Takeda
Tauhid Ali
rare disease
Oak Hill Bio
All Takeda wants for its billion-dollar gene therapy spending spree is functional cures
Fierce Biotech
Wed, 05/4/22 - 10:20 am
Takeda
gene therapy
rare disease
Selecta back in phase 1 gene therapy saddle after clinical hold lift, plans to reboot 'expeditiously'
Fierce Biotech
Thu, 03/10/22 - 10:27 pm
Selecta Biosciences
gene therapy
rare disease
methylmalonic acidemia
clinical trials
Novo makes a rare disease push
EP Vantage
Wed, 03/9/22 - 10:42 am
Novo Nordisk
rare disease
Rare disease and neurology take outs tick higher
EP Vantage
Wed, 03/2/22 - 10:03 am
M&A
AbbVie
AstraZeneca
Bristol Myers Squibb
JNJ
Gilead Sciences
Jazz Pharmaceuticals
Merck
Pfizer
Sanofi
rare disease
neurology
Kids suffer serious adverse reactions in AGTC gene therapy trial
Fierce Biotech
Fri, 09/24/21 - 12:04 pm
AGTC
gene therapy
pediatrics
clinical trials
rare disease
Martin Mackay’s rare disease startup gets $145 million as it enters the clinic
Endpoints
Tue, 05/19/20 - 10:42 am
Martin Mackay
Alexion
rare disease
Rallybio
FNAIT
RDMD raises $14M for rare disease real-world evidence generation
MedCity News
Fri, 04/17/20 - 08:55 pm
RDMD
startup
rare disease
RWE
real world evidence
Why So Many Biotechs Are Scrambling to Develop a Drug for the Same Rare Disease
Motley Fool
Sun, 02/23/20 - 06:17 pm
beta thalassemia
rare disease
drug development
Acceleron
Bristol-Myers Squibb
Bluebird Bio
Sangamo Therapeutics
Vertex Pharmaceuticals
Editas Medicine
Syros Pharmaceuticals
Healx Bags $56M to Advance Affordable, AI-Based Orphan Drugs
Xconomy
Fri, 10/18/19 - 10:20 am
Healx
rare disease
machine learning
artificial intelligence
orphan drug
How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025
Forbes
Fri, 11/2/18 - 09:18 am
Healx
rare disease
artificial intelligence
Eidos Therapeutics raises $106M IPO to advance ATTR med
Fierce Biotech
Wed, 06/20/18 - 03:41 pm
Eidos Therapeutics
IPOs
ATTR
rare disease
Pfizer's rare heart disease drug succeeds in late-stage study
Yahoo/Reuters
Fri, 03/30/18 - 09:35 am
Pfizer
clinical trials
tafamidis
transthyretin cardiomyopathy
rare disease
Alnylam
Ionis Pharmaceuticals
Shire CEO sees Microsoft tie-up building momentum for faster rare disease diagnosis
Fierce Pharma
Wed, 03/21/18 - 11:28 pm
Shire
Pharma CEOs
Microsoft
rare disease
Flemming Ornskov
Rare diseases: FDA awards grants over 6 million dollars
GoinPharma
Mon, 10/9/17 - 11:07 am
FDA
grants
rare disease

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