Latest News

Tuesday, June 30, 2015

Parents turn to data after son is diagnosed with ultra-rare disease
Scope (blog)
Four years ago, Matthew Might, PhD, and his wife, Christina, learned that their son Bertrand was the first person to be diagnosed with ultra-rare genetic disorder called N-Glycanase Disorder. At the 2015 Big Data in Biomedicine conference at Stanford ...

Monday, June 29, 2015

WIBW

Young Titan Rallies Against Rare Disease
WIBW
MANHATTAN, Kan. (WIBW) - Smiles and squeals fill the Manhattan home where four-year-old Avalyn Larson is playing with her 11-month-old brother, Titan. "He's always been very happy," dad Lee says of his younger child. "He loves his sister. His sister is ...

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Monday, June 29, 2015

CHMP Backs Three Drugs for Rare Diseases
Medscape
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing clearance of three drugs for rare diseases. The committee recommended approval of sebelipase alfa ( Kanuma , Synageva ...

Monday, June 29, 2015

Deadline

Gordon Gray's Race For Miracle Batten Disease Cure Update: Jon Hamm ...
Deadline
On June 9, Deadline revealed how Miracle producer Gordon Gray was racing the clock to raise a minimum of $10 million to run trials for a treatment that could possibly stop the progression of a rare degenerative brain disease in his two daughters.

Monday, June 29, 2015

Sports and Weather Right Now

Man Overcomes Rare Disease to Compete in IRONMAN
Sports and Weather Right Now
That's because 5 years ago, Ivan was diagnosed with scleroderma, a rare autoimmune disease. "At that point they told me I had 5 years to live, and I had to live with that for 6 months," said Ivan. "The hardest 6 months I'd ever gone through...I gained ...

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Monday, June 29, 2015

Daily Mail

Yasmin Butler whose face SUNK says she 'finally sees beauty in the mirror'
Daily Mail
Parry-Romberg syndrome is a rare disorder characterised by slowly progressive deterioration of the skin and soft tissues of half of the face, usually the left side. It is more common in females than males, and affects around 500 people around the world.

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Monday, June 29, 2015

WISH-TV

Family celebrates milestone birthday for son with rare disease
WISH-TV
GREENWOOD, Ind. (WISH) — A very special birthday for an Indiana boy whose family made national headlines in the fight against an extremely rare disease is Monday. 24-Hour News 8 first shared the story of the Milto family from Greenwood 15 years ago.

Saturday, June 27, 2015

European Pharmaceutical Review

EU Recommends Approval for Alexion's Rare Disease Drugs
Drug Discovery & Development
HPP is a genetic, progressive, ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to debilitating or life-threatening complications. It is characterized by defective bone ...
First Treatment Recommended For Rare Bone DiseaseLife Science Leader Magazine
Ten new medicines recommended in EuropePMLiVE
EMA Recommends First Hypophosphatasia MedicineMedscape
MarketWatch
all 16 news articles »
Saturday, June 27, 2015

Mirror.co.uk

Novak Djokovic: Boy with rare disease left delighted after star plays tennis ...
Mirror.co.uk
A brave little boy who suffers from a rare blood disease was left uttering "You cannot be serious" when he was treated to a tennis knock-up with Novak Djokovic. Jake Valentine, seven, couldn't believe his luck when he was invited on to the court for a ...

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Saturday, June 27, 2015

Irish Mirror

Novak Djokovic: Boy with rare disease left delighted after star plays tennis ...
Irish Mirror
A brave little boy who suffers from a rare blood disease was left uttering "You cannot be serious" when he was treated to a tennis knock-up with Novak Djokovic . Jake Valentine, seven, couldn't believe his luck when he was invited on to the court for a ...

and more »