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Rare Diseases

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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Biotech M&A is back. Ipsen set to fork out up to $1.3B for rare disease specialist Clementia

Biotech M&A is back. Ipsen set to fork out up to $1.3B for rare disease specialist Clementia

Endpoints

Clementia Pharmaceuticals

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China releases list of rare disease drugs with reduced VAT

China releases list of rare disease drugs with reduced VAT

BioCentury

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Pfizer, Merck, J&J well-positioned for M&A and Biogen and BioMarin are prime targets: report

Pfizer, Merck, J&J well-positioned for M&A and Biogen and BioMarin are prime targets: report

Fierce Pharma

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China to Cut Value Added Tax on Rare Disease Drugs to 3%

China to Cut Value Added Tax on Rare Disease Drugs to 3%

CP Wire

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Sanofi Cuts a Whopping 38 R&D Programs to Refocus on Cancer, Immunology, and Rare Diseases

Sanofi Cuts a Whopping 38 R&D Programs to Refocus on Cancer, Immunology, and Rare Diseases

BioSpace

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