Rare Diseases

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 2017-09-25 11:00 

Forbes

In A First, Pfizer Spins Out Biotech Firm With $103 Million In Funding... +

Pfizer is announcing today that it is spinning out a new company, SpringWorks Therapeutics, to develop several drugs for rare diseases that the drug giant did not want to move forward internally....

 2017-09-20 19:11 

Reuters

Novartis's Rydapt wins EU approval for AML, other diseases... +

Swiss drugmaker Novartis on Wednesday won European Union approval for Rydapt to be used against a mutated form of acute myeloid leukemia (AML) and other rare diseases, adding to U.S. approvals the medicine secured in April....

 2017-09-20 15:56 

The Weekly Standard

The FDA Old Guard Backtracks on Rare Diseases—Again... +

Michael AstrueOnce again officials within the FDA are proposing to put other interests over the needs of dying patients, predominantly children. For more than three decades a group of FDA civil servants aligned with Ralph Naders Public Citizen has fought ...

 2017-09-20 05:32 

Reuters

Novartis's Rydapt wins EU approval for AML, other diseases... +

Swiss drugmaker Novartis on Wednesday said it had won European Union approval for Rydapt to be used against a mutated form of acute myeloid leukemia (AML) and other rare diseases, adding to U.S. approvals it secured in April....

 2017-09-20 05:31 

Reuters

Novartis's Rydapt wins EU approval for AML, other diseases... +

Swiss drugmaker Novartis on Wednesday said it had won European Union approval for Rydapt to be used against a mutated form of acute myeloid leukemia (AML) and other rare diseases, adding to U.S. approvals it secured in April....

 2017-09-19 21:41 

Wall Street Journal

A Positive Medical Lesson From a Tragic Early Death... +

Patients living with rare diseases face a classic chicken-and-egg problem because without public awareness, building support for needed research and treatments remains an uphill struggle...

 2017-09-13 17:20 

NPR

FDA Moves To Rein In Drugmakers' Abuse Of Orphan Drug Law... +

Following an investigation by Kaiser Health News and NPR, the Food and Drug Administration has moved to close a loophole that let drugmakers skip pediatric testing of drugs to treat rare diseases....

 2017-09-12 20:29 

Reuters

BRIEF-Inotek Pharmaceuticals announces merger agreement with Rocket Pharmaceuticals to advance pipeline of first-in-class gene therapies for rare diseases... +

Inotek Pharmaceuticals Corp...

 2017-09-12 14:01 

The Huffington Post

IBM Watson Plays Know-It-All at US Tennis Open... +

IBMs Watson eats data for breakfast, lunch and dinner. Weve seen it play chess, diagnose rare diseases, drive cars,......

 2017-09-09 18:01 

Express

Iron deficiency warning: Could YOU spot symptoms of these three RARE diseases?... +

IRON deficiency is one of the possible causes of a rare disease called pica syndrome where sufferers eat everything from paint to faeces....

 2017-09-01 07:00 

Vancouver Sun

Rare diseases affect 1 in 3 children admitted to BC Childrens Hospital... +

Hours after she was born in Penticton in May 2015, Charlotte Palmer was flown to BC Childrens Hospital. Just 29 weeks old, baby Charlotte went on to endure months of infections and breathing probl...

 2017-08-30 10:14 

Malay Mail Online

Bringing hope to people living with rare diseases Azrul Mohd Khalib... +

AUGUST 30 The politics surrounding healthcare issues are often extremely diverse, divisive and complex. All we need to do is look at the United States ongoing debate regarding the Affordable Care Act, the United Kingdoms reform of its...

 2017-08-28 17:31 

Fox 5 NY

Kids battling rare diseases become superheroes... +

A 5-year-old boy battling neuroblastoma had the chance to become Batman for a day, thanks to a moving photo shoot that transformed cancer-stricken kids into superheroes....

 2017-08-28 11:00 

VentureBeat

Sequoia co-leads $30 million investment in MedGenomes research and diagnostics testing... +

MedGenome wants to develop research and diagnostics around rare diseases by looking at Indias population of 1.3 billion and today announced funding of $30 million to further its mission. Sequoia Capital India and Sofina co-led the round, with participatio...

 2017-08-26 15:46 

The Telegraph

How do you turn world-leading British science into medicines?... +

It may not be glamorous, but Stevenage is set to become a world-leading centre for gene therapies, the new wave of pioneering treatments that hold great promise for tackling illnesses such as cancer and rare diseases....

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