Latest News

Friday, April 28, 2017

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 ...

Friday, April 28, 2017

Fortune

The FDA Just Approved One of the Most Expensive Drugs in the World
Fortune
It's the first-ever drug approved for a form of Batten disease, a rare genetic disorder that ravages the nervous system and can cause symptoms ranging from seizures to trouble coordinating muscles to vision loss. It can affect both adults and children ...
New drug for rare disease wins approval, a "comfort" for family too late to benefitCBS News
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
The Commercial Appeal -Reuters -BioPharma Dive -FDA.gov
all 42 news articles »
Friday, April 28, 2017

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

all 3 news articles »
Thursday, April 27, 2017

Endpoints News

UPDATED: BioMarin scores a quick FDA OK for its latest rare disease entry — flagged at $702K WAC
Endpoints News
BioMarin $BMRN attracted attention for its latest rare disease drug for its efficacy in treating Neuronal Ceroid Lipofuscinosis Type 2 disease (CLN2), a form of Batten disease. The disease progresses rapidly, with recurrent seizures and vision loss ...
BREAKING NEWS: FDA Approves BioMarin's Batten Disease DrugRare Disease Report
Biomarin sets high price for rare pediatric brain disease drugReuters
New drug to treat Batten disease OK'd but won't help Gray girlsChicago Tribune
News-Medical.net -Xconomy
all 24 news articles »
Thursday, April 27, 2017

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 ...

and more »
Thursday, April 27, 2017

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.

Thursday, April 27, 2017

New York Times

Life with a Rare Genetic Disease: The Science, the Suffering and the Hope
New York Times
If life can be a Hobbesian trial — “nasty, brutish and short” — then for adults with rare genetic diseases, life can sometimes be nasty, brutish and long. Take the ordinary difficulties of getting through the day and add, for example, loss of motor ...

Thursday, April 27, 2017

Coffs Coast Advocate

Mother's plea to save baby boy dying from rare disease
Coffs Coast Advocate
Baby Peter has been diagnosed with a rare and complex congenital heart condition which needs to be corrected urgently, but because the Solomon Islands is a developing country there is no access to cardiologists that can perform the interventionist surgery.

Wednesday, April 26, 2017

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 ...

Wednesday, April 26, 2017

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.

and more »