Latest News

Sunday, May 24, 2015

Shiprock toddler with rare disease receives liver transplant in California
Farmington Daily Times
When Kaiden was 14 months old, he was diagnosed with a rare genetic condition known as mitochondrial neurogastrointestinal encephalopathy, or MNGIE. The condition affects his ability to absorb nutrients and move food through his body. Charley and ...

Sunday, May 24, 2015

St George News

Hatch, Klobuchar propose law to foster development of rare disease treatments
St George News
The “OPEN ACT,” as it is being called, would build on the success of the Orphan Drug Act to provide incentives to increase market potential for companies that “repurpose” previously-approved drugs for the treatment of rare diseases. Repurposing drugs ...

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Sunday, May 24, 2015

Sports-mad Zac bounces back to win race after rare disease battle
Clacton and Frinton Gazette
A SPORTS-MAD youngster who was left fighting for his life in a hospital bed after catching a rare disease has bounced back to win his school's Race For Life. Zac Mathews was just six when he was taken to Colchester General Hospital suffering from ...

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Sunday, May 24, 2015

SFGate

Mongolian girl battles rare disease, separation from mother
SFGate
It's an escape from the one-bedroom apartment they share with four Mongolian men while Ganbat's daughter Nomin, 4, undergoes experimental treatment for a rare brain disease at UCSF Benioff Children's Hospital Oakland. They've been in Oakland since ...

Friday, May 22, 2015

News-Medical.net

Lafora rare disease could be aggravated by oxidative stress
News-Medical.net
Researchers at the University of Valencia have led a study in which they propose that Lafora rare disease could be aggravated by oxidative stress. These ideas have been put forward in a review article recently published in the journal Free Radical ...

Friday, May 22, 2015

Illawarra Mercury

Illawarra siblings rare disorder leads photo exhibition
Illawarra Mercury
Illawarra kids Madeleine Saxby, Kristian Krstevski and Kate Hinchcliff all have something in common - they each have a sibling who suffers from a rare disease. The three have united for a photographic project called The Forgotten Ones which was ...

Friday, May 22, 2015

Clinical trials for rare diseases – finding and keeping patients
ScienceBlog.com (blog)
Clinical trials are essential to ensure drug safety and efficacy, and the recent increase in the development of orphan drugs has led to an increase in rare disease clinical trials. The nature of rare diseases creates specific challenges for clinical ...

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Thursday, May 21, 2015

NZORD - the New Zealand Organisation for Rare Disorders
Scoop.co.nz
Yesterday, founder and Executive Director, John Forman, announced that he is stepping down from his current position with The New Zealand Organisation for Rare Disorders. NZORD is a not-for-profit that exists to inform and support rare disease families ...

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Thursday, May 21, 2015

ABC NEWS 4

Woman with rare blood disease needs a transfusion every three weeks to stay ...
ABC NEWS 4
Imagine needing a blood transfusion monthly to stay alive: That's the case for 44-year-old Laurie Shore. “If I couldn't get blood that's it,” Shore said. “I'd go into a coma or death.” Shore lives with a rare disease called Diamond Black Fan Anemia. It ...

Thursday, May 21, 2015

Advocates Cheer Senate Leaders for Introducing Bipartisan Bill to Increase ...
PR Newswire (press release)
Supported by more than 150 rare disease patient advocacy organizations, this bipartisan legislation promises to rapidly bring hundreds of safe, effective, and affordable medicines to rare disease patients by incentivizing drug makers to "repurpose ...