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Rare Diseases
How Covid-19 is changing rare diseases research
How Covid-19 is changing rare diseases research
MedCity News
COVID-19
Rare Diseases
R&D
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Pharmacquired: What a surprise deal does for AstraZeneca and Alexion
Pharmacquired: What a surprise deal does for AstraZeneca and Alexion
BioPharma Dive
AstraZeneca
Alexion
M&A
Rare Diseases
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AstraZeneca to buy Alexion for $39B
AstraZeneca to buy Alexion for $39B
BioPharma Dive
AstraZeneca
Alexion
M&A
Rare Diseases
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FDA to expedite development of two Sanofi rare disease drugs
FDA to expedite development of two Sanofi rare disease drugs
Pharmaforum
Sanofi
FDA
Rare Diseases
Pompe disease
Avalglucosidase alfa
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Takeda drops $300M cash to kick off $1B RNAi alliance with Arrowhead, shooting for first-in-class rare disease drug
Takeda drops $300M cash to kick off $1B RNAi alliance with Arrowhead, shooting for first-in-class rare disease drug
Endpoints
Takeda
RNAi
Arrowhead Pharmaceuticals
Rare Diseases
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FDA seeks input on rare disease clinical trials network
FDA seeks input on rare disease clinical trials network
RAPS.org
FDA
Rare Diseases
clinical trials
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With money flowing, Pliant Therapeutics heads to public well
With money flowing, Pliant Therapeutics heads to public well
Endpoints
IPOs
Pliant Therapeutics
Rare Diseases
oncology
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Takeda signs $882m rare disease alliance with Oxford biotech Evox
Takeda signs $882m rare disease alliance with Oxford biotech Evox
Pharmaforum
Evox Therapeutics
Takeda
Rare Diseases
biotech
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The biggest challenges facing rare disease pharma – and how the industry can tackle them
The biggest challenges facing rare disease pharma – and how the industry can tackle them
Pharmaforum
Rare Diseases
orphan drugs
drug development
FDA
drug pricing
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Spark, Ipsen and more join advocacy groups, agencies in Rare Disease Day 2020 efforts
Spark, Ipsen and more join advocacy groups, agencies in Rare Disease Day 2020 efforts
Fierce Pharma
Spark Therapeutics
Ipsen
Rare Diseases
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Regeneron's bone disease drug delivers in phase 2, poised for FDA filing
Regeneron's bone disease drug delivers in phase 2, poised for FDA filing
Fierce Biotech
Regeneron
Rare Diseases
FDA
clinical trials
garetosmab
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3 Pharma & Biotech Pipeline Areas to Keep an Eye on in 2020
3 Pharma & Biotech Pipeline Areas to Keep an Eye on in 2020
Yahoo/Zacks.com
Alzheimer's disease
Medical Marijuana
Rare Diseases
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Drugmakers rebranding around oncology, rare diseases could provide 2020 preview
Drugmakers rebranding around oncology, rare diseases could provide 2020 preview
Fierce Pharma
oncology
Rare Diseases
AstraZeneca
GSK
Alexion
Takeda
Soliris
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Horizon looks to the future with massive San Francisco manufacturing, R&D plant
Horizon looks to the future with massive San Francisco manufacturing, R&D plant
Fierce Pharma
Horizon Therapeutics
eye health
R&D
drug manufacturing
Rare Diseases
San Francisco
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Takeda sees cell, gene therapy in its future. Is it too late?
Takeda sees cell, gene therapy in its future. Is it too late?
Biopharma Dive
Takeda
gene therapy
Rare Diseases
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Rare Disease Clinical Trials: FDA Awards $15M in Grants
Rare Disease Clinical Trials: FDA Awards $15M in Grants
RAPS.org
grants
clinical trials
FDA
Rare Diseases
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Rare genetic disease company Homology raises $125M follow-on
Rare genetic disease company Homology raises $125M follow-on
BioCentury
Homology Med
Rare Diseases
gene therapies
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Industry and Advocacy Groups Urge FDA to Allow More Real-World Data for Rare Disease Trials
Industry and Advocacy Groups Urge FDA to Allow More Real-World Data for Rare Disease Trials
BioSpace
FDA
Rare Diseases
real world data
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How pharma can support doctors who treat rare diseases
How pharma can support doctors who treat rare diseases
Pharmaforum
physicians
Rare Diseases
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Rare Disease Day: How the patient voice can tear down barriers
Rare Disease Day: How the patient voice can tear down barriers
Pharmaforum
patients
Rare Diseases
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