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Rare Diseases

GSK slims portfolio with sale of rare disease gene therapy drugs

GSK slims portfolio with sale of rare disease gene therapy drugs

Yahoo/Reuters

Orchard Therapeutics

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Alexion buying Swedish biotech for $855 million to broaden rare disease pipeline

Alexion buying Swedish biotech for $855 million to broaden rare disease pipeline

Stat

WIlson Therapeutics

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On Rare Disease Day, recognizing the life and work of biotech giant Henri A. Termeer

On Rare Disease Day, recognizing the life and work of biotech giant Henri A. Termeer

Endpoints

Rare Disease Day

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Idera, Biocryst to merge, rebrand and focus in rare disease programs

Idera, Biocryst to merge, rebrand and focus in rare disease programs

Endpoints

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Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss

Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss

RAPS.org

priority review vouchers

drug development

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More efficient rare pediatric drug development: FDA drafts guidance

More efficient rare pediatric drug development: FDA drafts guidance

Endpoints

drug development

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Alexion bolsters Soliris patent defense

Alexion bolsters Soliris patent defense

BioPharma Dive

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5 Trends influencing drug pricing

5 Trends influencing drug pricing

BioPharma Dive

drug development

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GlaxoSmithKline's talk of leaving rare diseases—amid slow Strimvelis sales—highlights challenges in the field

GlaxoSmithKline's talk of leaving rare diseases—amid slow Strimvelis sales—highlights challenges in the field

Fierce Pharma

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FDA Reverses Decision, Allowing Amicus To File For Rare Disease Drug

FDA Reverses Decision, Allowing Amicus To File For Rare Disease Drug

Forbes

Amicus Therapeutics

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Nearly every $100,000 drug approved in the last three years was for a rare disease

Nearly every $100,000 drug approved in the last three years was for a rare disease

Medical Marketing and Media

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Tapping into the rare disease market

Tapping into the rare disease market

BioPharma Dive

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Amicus CEO John Crowley, Not His Daughter Megan, Might Compel Trump to Make Big Changes at FDA

Amicus CEO John Crowley, Not His Daughter Megan, Might Compel Trump to Make Big Changes at FDA

TheStreet.com

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Trump administration raises questions for rare-disease drugmakers

Trump administration raises questions for rare-disease drugmakers

Medical Marketing and Media

Affordable Care Act

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FDA awards 21 grants to stimulate product development for rare diseases

FDA awards 21 grants to stimulate product development for rare diseases

FDA.gov

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Horizon Buys Rare-Disease Company Raptor for $800 Million

Horizon Buys Rare-Disease Company Raptor for $800 Million

Bloomberg

Raptor Pharmaceutical

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Shire PLC (ADR) May Now Seek Rare Disease Expansion With Raptor Pharmaceutical Corp.

Shire PLC (ADR) May Now Seek Rare Disease Expansion With Raptor Pharmaceutical Corp.

Bidness, ETC

Raptor Pharmaceuticals

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FDA should listen to patients, not just clinical trial data, on rare disease therapies

FDA should listen to patients, not just clinical trial data, on rare disease therapies

Stat

orphan diseasese

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Shire and Cincinnati Children's Hospital team up in rare diseases

Shire and Cincinnati Children's Hospital team up in rare diseases

Medical Marketing and Media

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FDA's rare disease pediatric voucher program could become permanent

FDA's rare disease pediatric voucher program could become permanent

BioPharma Dive

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