Rare Diseases

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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Takeda, Microsoft rare disease partnership publishes diagnosis roadmap
Takeda, Microsoft rare disease partnership publishes diagnosis roadmap
Fierce Pharma
Takeda
Microsoft
Eurodis
diagnosis
Rare Diseases
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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
M&A
Ipsen
Clementia Pharmaceuticals
Rare Diseases
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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
China
VAT
Rare Diseases
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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
M&A
oncology
neurology
Rare Diseases
Pfizer
Merck
JNJ
Biogen
Biomarin
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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
China
Rare Diseases
VAT
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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
Sanofi
Rare Diseases
immunology
cancer
R&D
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Clinical Costs for Orphan Drugs Lower Than Non-Orphan Drugs, Study Finds
Clinical Costs for Orphan Drugs Lower Than Non-Orphan Drugs, Study Finds
RAPS.org
orphan drugs
Rare Diseases
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FDA Faulted For Lapses In Orphan Drug Program
FDA Faulted For Lapses In Orphan Drug Program
NPR
FDA
orphan drugs
Rare Diseases
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Should FDA Treat Rare Disease Drugs Differently?
Should FDA Treat Rare Disease Drugs Differently?
MedPage
FDA
patients
Rare Diseases
orphan diseases
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Proposal seeks FDA center for rare diseases
Proposal seeks FDA center for rare diseases
BioCentury
FDA
Rare Diseases
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An Unwarranted Attack On Rare Disease Research
An Unwarranted Attack On Rare Disease Research
Forbes
R&D
Rare Diseases
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Rare Disease Biotechs Avrobio, Eidos Therapeutics File for IPOs
Rare Disease Biotechs Avrobio, Eidos Therapeutics File for IPOs
Xconomy
Avrobio
Eidos Therapeutics
Rare Diseases
IPOs
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GSK slims portfolio with sale of rare disease gene therapy drugs
GSK slims portfolio with sale of rare disease gene therapy drugs
Yahoo/Reuters
GSK
Orchard Therapeutics
gene therapy
Rare Diseases
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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
Alexion
WIlson Therapeutics
M&A
Rare Diseases
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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 Diseases
Rare Disease Day
biotech
Henri Termeer
Genzyme
Sanofi
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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
Idera
Biocryst
M&A
Rare Diseases
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