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Wednesday, July 30, 2014

Cantech Letter

BioMarin sells special FDA voucher for $67.5 million
SFGate (blog)
Back in February, BioMarin won a voucher from the U.S. Food and Drug Administration that is intended to encourage the development of treatments for rare pediatric diseases. It entitles the voucher's holder to an expedited FDA review of a new drug, ...
Sanofi, Regeneron pay $67.5M for a regulatory shortcut in blockbuster PCSK9 ...FierceBiotech
BioMarin Sells Priority Review Voucher for $67.5 MillionNASDAQ
Regeneron and Sanofi Announce Plan to Use Priority Review Voucher For ...Wall Street Journal

all 14 news articles »
Wednesday, July 30, 2014

Reuters

BioMarin Sells Priority Review Voucher for $67.5 Million
MarketWatch
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that it has sold the Rare Pediatric Disease Priority Review Voucher (PRV) it obtained in February of this year. The Company received the voucher under an FDA program intended to ...
BioMarin sells special FDA voucher for $67.5 millionSFGate (blog)
Sanofi, Regeneron pay $67.5M for a regulatory shortcut in blockbuster PCSK9 ...FierceBiotech
Biotechs to use priority review voucher for cholesterol-lowering drug candidateSeeking Alpha (registration)

all 41 news articles »
Wednesday, July 30, 2014

After Discovering Rare Disease Isaiah Austin Gets Job With NBA [VIDEO]
Classicalite
Sadly, however, just before the draft, Austin discovered he had Marfan Syndrome, a rare genetic disorder that can be fatal in extreme cases. Since he can no longer compete, NBA Commissioner instead has offered him a job with the NBA. He plans to stay ...

Wednesday, July 30, 2014

FDA Boosts Sarepta With Prognosis for its Rare Disease Drug
Wall Street Journal (blog)
A top FDA official says the agency is exploring all “potential pathways” to approve new drugs to treat the rare childhood disease, which causes muscles in boys to stop working and eventually results in death because they can no longer breathe. “We ...

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Wednesday, July 30, 2014

Irish Independent

Mum of tragic boy (4) with rare disease struggles to raise €10k for necessary ...
Irish Independent
He has open wounds as a result of a condition deterioration last January and suffers night-time seizures. Adam was born in 2010 with a a twisted bowl and a heart irregularity - his heart was beating too fast. His parents were told he 'probably wouldn't ...

Tuesday, July 29, 2014

Rare disease like breathing through a straw
Windsor Star (blog)
Anderson has a rare and terminal disease called Alpha-1 antitrypsin deficiency. It is often mistaken for asthma or COPD. Tape your nostrils shut and breathe through a straw to get an idea of what it feels like, she said. Anderson has survived past her ...

Monday, July 28, 2014

Rare diseases drug plan announced for New Brunswick
CBC.ca
Some New Brunswickers with rare diseases will now have help with the high associated drug costs. The provincial government announced the New Brunswick Drugs for Rare Diseases Plan on Monday. It will cover the cost of five drugs for specific diseases at ...
New Brunswick announces plan to pay for drugs to treat rare diseasesmysask.com

all 6 news articles »
Monday, July 28, 2014

NB sets up fund for rare disease drug costs
TheChronicleHerald.ca
FREDERICTON — A program has been launched in New Brunswick to help people pay for drugs for rare diseases. The plan will cover the costs of five drugs for rare diseases including Hunter syndrome and Pompe disease. Health Minister Hugh Flemming ...
Rare diseases drug plan announced for New BrunswickCBC.ca

all 6 news articles »
Sunday, July 27, 2014

WRGB

Local boy raises money and awareness on rare disease
WRGB
This year he's paying it forward by raising awareness for the disease. He's having a bottle and can drive. They've already raised plenty. They've well exceeded their goal of $1000 and now they have a new goal of $2000, one you can help him reach.

Sunday, July 27, 2014

The Press

Family on constant vigil with rare disease
The Press
One of the few genetic scientists dedicated to researching the rare motor neuron disease, American Greg Fox, wrote that "as genetic diseases go, SMARD is the rarest of the rare". It involves progressive destruction of cells which control motor ...