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Wednesday, October 01, 2014

Boston Business Journal (blog)

Zafgen begins late-stage study of obesity drug in rare-disease patients
Boston Business Journal (blog)
A late-stage trial of a potential drug to treat obesity by Zafgen will enroll 84 adolescents and adults across the U.S. with a rare disease known as Prader-Willi syndrome. The trial, which kicked off today, will test the Boston-based biotech firm's ...
Zafgen Announces Initiation of Phase 3 Trial of Beloranib in Prader-Willi SyndromeNASDAQ

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Wednesday, October 01, 2014

Alnylam launches study to support its rare disease candidate ALN-AS1
FierceDrugDelivery
RNAi leader Alnylam ($ALNY), the American Porphyria Consortium and The European Porphyria Network are launching the Explore observational study of patients with the rare disease, with the hope of advancing Alnylam's candidate for porphyria, with a ...

Wednesday, October 01, 2014

FDA awards $19m in rare disease grants
PMLiVE
“The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program,” said Dr Gayatri Rao, director of the ...
FDA grants to stimulate drug and device development for rare diseasesThe Pharma Letter

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Tuesday, September 30, 2014

Patients With Rare Diseases Hold Rally
The New Indian Express
There are about 50 known patients in the state suffering from LSDs, a group of over 45 rare genetic disorders that occur due to deficiency of specific enzymes in special compartments (lysosomes) of cells. Prasanna Shirol, founder member, Organisation ...

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Tuesday, September 30, 2014

Parents Of 3-Year-Old Girl With Rett Syndrome Raising Awareness Of Rare ...
CBS Local
(CBS) — It's a rare disorder with no cure and it strikes without warning, robbing little girls of their ability to walk, talk, eat and even breathe. In this Original Report, CBS 2's Mai Martinez takes a look at Rett syndrome and one Chicago family's ...

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Tuesday, September 30, 2014

Fundraiser in West Haven to raise funds for baby with rare disease
New Haven Register
Fundraiser in West Haven to raise funds for baby with rare disease. Colton Amendola. Contributed photo. Posted: 09/30/14, 4:40 PM EDT |. # Comments. Colton Amendola. Contributed photo. WEST HAVEN >> Family and supporters of Colton Amendola will ...

Tuesday, September 30, 2014

FDA awards grants to stimulate drug, device development for rare diseases
FDA.gov
The U.S. Food and Drug Administration today announced it has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the ...

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Tuesday, September 30, 2014

Amicus Therapeutics to Present at Leerink Partners Rare Disease Roundtable
CNNMoney
30, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today announced that Bradley Campbell, Chief Operating Officer and Chip Baird, Chief Financial Officer, will present a corporate overview at the Leerink Partners Rare Disease ...

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Tuesday, September 30, 2014

Corbus Pharmaceuticals to Present at the Leerink Partners Rare Disease ...
Marketwired (press release)
NORWOOD, MA--(Marketwired - Sep 30, 2014) - Corbus Pharmaceuticals Holdings, Inc., an emerging drug development company focused on the development and commercialization of its lead product candidate, Resunab™ -- for the treatment of rare, ...

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Tuesday, September 30, 2014

Catalyst Pharma Closer to Unconscionable Price Hike for Rare-Disease Drug
TheStreet.com
Catalyst reported positive results from a phase III study of Firdapse as a treatment for Lambert-Eaton Myasthenic Syndrome (LEMS), a progressive, muscle-weakening disease. Treatment with Firdapse improved LEMS symptoms compared to a placebo.
Catalyst soars as its controversial rare disease drug aces Phase IIIFierceBiotech
Catalyst Surges On Positive Trial ResultsBidness ETC

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