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Rare Diseases

Pharmacquired: What a surprise deal does for AstraZeneca and Alexion

Pharmacquired: What a surprise deal does for AstraZeneca and Alexion

BioPharma Dive

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AstraZeneca to buy Alexion for $39B

AstraZeneca to buy Alexion for $39B

BioPharma Dive

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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

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

Arrowhead Pharmaceuticals

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FDA seeks input on rare disease clinical trials network

FDA seeks input on rare disease clinical trials network

RAPS.org

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

Pliant Therapeutics

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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

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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

drug development

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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

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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

clinical trials

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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

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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

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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

drug manufacturing

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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

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Rare Disease Clinical Trials: FDA Awards $15M in Grants

Rare Disease Clinical Trials: FDA Awards $15M in Grants

RAPS.org

clinical trials

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Rare genetic disease company Homology raises $125M follow-on

Rare genetic disease company Homology raises $125M follow-on

BioCentury

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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

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

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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

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Takeda, Microsoft rare disease partnership publishes diagnosis roadmap

Takeda, Microsoft rare disease partnership publishes diagnosis roadmap

Fierce Pharma

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