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» Rare Diseases
Rare Diseases
Industry and Advocacy Groups Urge FDA to Allow More Real-World Data for Rare Disease Trials
BioSpace
Fri, 04/5/19 - 11:48 am
FDA
Rare Diseases
real world data
How pharma can support doctors who treat rare diseases
Pharmaforum
Thu, 04/4/19 - 11:14 pm
physicians
Rare Diseases
Rare Disease Day: How the patient voice can tear down barriers
Pharmaforum
Thu, 02/28/19 - 09:49 am
patients
Rare Diseases
Takeda, Microsoft rare disease partnership publishes diagnosis roadmap
Fierce Pharma
Wed, 02/27/19 - 11:52 am
Takeda
Microsoft
Eurodis
diagnosis
Rare Diseases
Biotech M&A is back. Ipsen set to fork out up to $1.3B for rare disease specialist Clementia
Endpoints
Mon, 02/25/19 - 10:04 am
M&A
Ipsen
Clementia Pharmaceuticals
Rare Diseases
China releases list of rare disease drugs with reduced VAT
BioCentury
Sun, 02/24/19 - 11:24 pm
China
VAT
Rare Diseases
Pfizer, Merck, J&J well-positioned for M&A and Biogen and BioMarin are prime targets: report
Fierce Pharma
Wed, 02/20/19 - 11:59 am
M&A
oncology
neurology
Rare Diseases
Pfizer
Merck
JNJ
Biogen
Biomarin
China to Cut Value Added Tax on Rare Disease Drugs to 3%
CP Wire
Wed, 02/13/19 - 10:02 am
China
Rare Diseases
VAT
Sanofi Cuts a Whopping 38 R&D Programs to Refocus on Cancer, Immunology, and Rare Diseases
BioSpace
Thu, 02/7/19 - 11:05 am
Sanofi
Rare Diseases
immunology
cancer
R&D
Clinical Costs for Orphan Drugs Lower Than Non-Orphan Drugs, Study Finds
RAPS.org
Sat, 01/12/19 - 11:33 am
orphan drugs
Rare Diseases
FDA Faulted For Lapses In Orphan Drug Program
NPR
Sat, 12/1/18 - 11:53 am
FDA
orphan drugs
Rare Diseases
Should FDA Treat Rare Disease Drugs Differently?
MedPage
Sat, 10/6/18 - 12:53 pm
FDA
patients
Rare Diseases
orphan diseases
Proposal seeks FDA center for rare diseases
BioCentury
Thu, 09/13/18 - 09:36 am
FDA
Rare Diseases
An Unwarranted Attack On Rare Disease Research
Forbes
Tue, 08/28/18 - 09:59 am
R&D
Rare Diseases
Rare Disease Biotechs Avrobio, Eidos Therapeutics File for IPOs
Xconomy
Sat, 05/26/18 - 09:22 am
Avrobio
Eidos Therapeutics
Rare Diseases
IPOs
GSK slims portfolio with sale of rare disease gene therapy drugs
Yahoo/Reuters
Thu, 04/12/18 - 12:02 pm
GSK
Orchard Therapeutics
gene therapy
Rare Diseases
Alexion buying Swedish biotech for $855 million to broaden rare disease pipeline
Stat
Wed, 04/11/18 - 11:10 am
Alexion
WIlson Therapeutics
M&A
Rare Diseases
On Rare Disease Day, recognizing the life and work of biotech giant Henri A. Termeer
Endpoints
Wed, 02/28/18 - 11:25 am
Rare Diseases
Rare Disease Day
biotech
Henri Termeer
Genzyme
Sanofi
Idera, Biocryst to merge, rebrand and focus in rare disease programs
Endpoints
Mon, 01/22/18 - 10:56 am
Idera
Biocryst
M&A
Rare Diseases
Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss
RAPS.org
Sat, 12/9/17 - 02:27 pm
priority review vouchers
Rare Diseases
R&D
drug development
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