Rare Disease in the News

CBS News

New drug for rare disease wins approval, a "comfort" for family too late to benefit
CBS News
"The days are long, but the years are short." That's how Jennifer and Tracy VanHoutan, the parents of Noah and Laine and Emily, describe what it's like to have a child with Batten disease, a rare and devastating neurological condition that took their ...
The FDA Just Approved One of the Most Expensive Drugs in the WorldFortune
UPDATED: BioMarin scores a quick FDA OK for its latest rare disease entry — flagged at $702K WACEndpoints News
FDA Approves BioMarin's Batten Disease Drug. Cost Per Year is $702000Rare Disease Report
Reuters -BioPharma Dive -Chicago Tribune -FDA.gov
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FierceBiotech

Takeda, Harrington set up rare disease research program ...
FierceBiotech
Takeda Pharmaceutical has teamed up with Harrington Discovery Institute to support research into treatments for rare diseases. The alliance builds on ...
Harrington Discovery Institute Teams with Takeda for Advancement ...Rare Disease Report

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Boston Business Journal

​Hyde Park boy with rare disease wins appeal, will receive Biogen drug
Boston Business Journal
Tyler Hansen has spinal muscular atrophy, a very rare disease that that reduces ability to control voluntary muscle movement. The first-ever drug to treat the disease, called Spinraza and marketed by Cambridge-based Biogen (Nasdaq: BIIB), was just ...


Fortune

Sanofi May Want to Buy Sarepta as CEO Set to Depart | Fortune.com
Fortune
French pharma giant Sanofi has been trying its best to snap up assets in the face of falling sales and competition for its flagship diabetes drug franchise.
Sarepta CEO to Resign after Company Wins FDA's First Approval for ...Genetic Engineering & Biotechnology News

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Tallahassee.com

Karlos Williams' son battles rare disease, fundraiser started
Tallahassee.com
Little Kason Williams was a happy, energetic child. But after only eight months of being in the world, his health took a downturn. "It was becoming so bad — he was just sick and lifeless. He didn't want to do anything but just lay on you, and that's ...


WABC-TV

Teen with rare disease gets special trip to Disney Store in Times Square thanks to NYPD
WABC-TV
They got a tour, some time to shop, and then they opened the store to the public. Jacob has Mitochondrial Disease, which prevents his body from producing energy. "Gratitude is too light of a word, it means the world to him," said Tom Priestly, Jacob's ...


Seeking Alpha

BioMarin Races Towards Next Rare Disease Approval - BioMarin ...
Seeking Alpha
BioMarin could receive FDA approval on Thursday for Brineura, a treatment for CLN2 disease. CLN2 disease is a rare genetic disease that strikes children and ...
Biomarin Newly Approved Drug for Rare, Childhood Disease Will ...TheStreet.com
BioMarin sees 'high end' pricing on Brineura ahead of FDA decision ...FiercePharma

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Kane County Chronicle

Fundraiser to shed light on rare disease community
Kane County Chronicle
CHARLES – Shannon Rowan understands how difficult it can be to navigate the rare disease community. The facts roll off her tongue freely: one in 10 Americans has a rare disease. Of the 7,000-plus known rare diseases, 95 percent do not have treatments.


Funds sought for Reno County woman struggling with rare disease
Hutchinson News
Porphyria is a rare disease with no bounds: suspected of driving King George III to madness and testing the will of a Reno County woman. Shirley Schrag, 66, was diagnosed in 2005 with acute intermittent porphyria, one of the eight forms of the disease ...


Takeda, Harrington Discovery Institute Launch Rare Disease ...
Genetic Engineering & Biotechnology News
Takeda Pharmaceutical will partner with Harrington Discovery Institute at University Hospitals in Cleveland on discoveries intended to advance development of ...

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