rare disease

Martin Mackay’s rare disease startup gets $145 million as it enters the clinic
Martin Mackay’s rare disease startup gets $145 million as it enters the clinic
Endpoints
Martin Mackay
Alexion
rare disease
Rallybio
FNAIT
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RDMD raises $14M for rare disease real-world evidence generation
RDMD raises $14M for rare disease real-world evidence generation
MedCity News
RDMD
startup
rare disease
RWE
real world evidence
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Why So Many Biotechs Are Scrambling to Develop a Drug for the Same Rare Disease
Why So Many Biotechs Are Scrambling to Develop a Drug for the Same Rare Disease
Motley Fool
beta thalassemia
rare disease
drug development
Acceleron
Bristol-Myers Squibb
Bluebird Bio
Sangamo Therapeutics
Vertex Pharmaceuticals
Editas Medicine
Syros Pharmaceuticals
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Healx Bags $56M to Advance Affordable, AI-Based Orphan Drugs
Healx Bags $56M to Advance Affordable, AI-Based Orphan Drugs
Xconomy
Healx
rare disease
machine learning
artificial intelligence
orphan drug
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How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025
How One Startup Is Using AI To Find Cures For 100 Rare Diseases By 2025
Forbes
Healx
rare disease
artificial intelligence
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Eidos Therapeutics raises $106M IPO to advance ATTR med
Eidos Therapeutics raises $106M IPO to advance ATTR med
Fierce Biotech
Eidos Therapeutics
IPOs
ATTR
rare disease
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Pfizer's rare heart disease drug succeeds in late-stage study
Pfizer's rare heart disease drug succeeds in late-stage study
Yahoo/Reuters
Pfizer
clinical trials
tafamidis
transthyretin cardiomyopathy
rare disease
Alnylam
Ionis Pharmaceuticals
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Shire CEO sees Microsoft tie-up building momentum for faster rare disease diagnosis
Shire CEO sees Microsoft tie-up building momentum for faster rare disease diagnosis
Fierce Pharma
Shire
Pharma CEOs
Microsoft
rare disease
Flemming Ornskov
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Rare diseases: FDA awards grants over 6 million dollars
Rare diseases: FDA awards grants over 6 million dollars
GoinPharma
FDA
grants
rare disease
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Alnylam’s rare disease drug shines in trial, paving way for a brand-new class of medicines
Alnylam’s rare disease drug shines in trial, paving way for a brand-new class of medicines
Stat
Alnylam
rare disease
Patisiran
familial amyloid polyneuropathy
RNAi
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Smaller sales forces get the job done in rare disease
Smaller sales forces get the job done in rare disease
BioPharma Dive
drug reps
sales force
rare disease
Taro Pharmaceuticals
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Rare obesity disease player Rhythm Pharma sets out in search of a $115M IPO windfall
Rare obesity disease player Rhythm Pharma sets out in search of a $115M IPO windfall
Endpoints
Rhythm Pharmaceuticals
IPOs
obesity
rare disease
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Rare-Disease Drugmakers Join in Worries Over GOP Health Overhaul
Rare-Disease Drugmakers Join in Worries Over GOP Health Overhaul
Bloomberg
rare disease
healthcare reform
Shire
Jazz Pharmaceuticals
Sanofi
Roche
Biogen
Alexion
Vertex Pharmaceuticals
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Shire, armed with new rare disease patent, aims to block CSL's Haegarda launch
Shire, armed with new rare disease patent, aims to block CSL's Haegarda launch
Fierce Pharma
Shire
rare disease
CSL
patents
Haegarda
HAE
hereditary angiodema
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Top 25 rare disease brands, by sales
Top 25 rare disease brands, by sales
Medical Marketing and Media
rare disease
Copaxone
Teva Pharmaceutical
Avonex
Biogen
Sensipar
Soliris
Alexion
Xyrem
Jazz Pharmaceuticals
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Pfizer’s Metabolic, Rare Disease Segments: Hope for the Future?
Pfizer’s Metabolic, Rare Disease Segments: Hope for the Future?
Market Realist
Pfizer
metabolic disorders
rare disease
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4 ways for marketers to differentiate their brands in the rare-disease space
4 ways for marketers to differentiate their brands in the rare-disease space
Medical Marketing and Media
rare disease
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Drugs for rare diseases looming health hazard
Drugs for rare diseases looming health hazard
Toronto Star, Canada
orphan drugs
rare disease
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