Rare Disease in the News

3-year-old's battle against rare disease changing medicine, helping others
WGAL Lancaster
The procedure went well and the rare disease that didn't allow Kennedy's body to process protein has now been cured. Not only is Kennedy able to eat foods she couldn't before, other developmental problems seem to be improving as well. Since Kennedy is ...


Andrea Kruger's Mother Gets Amputations After Rare Disease
Ryan Roberts said it happened fast. "Within 12 hours she pretty much became non-responsive" said Ryan. Teri Roberts came to Methodist Woman's Hospital on December 10th, after only a few days of feeling sick. But within hours of her arrival to the ...

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Pittsburgh Post Gazette

Fighting rare disease, Hampton second-grader finds acceptance
Pittsburgh Post Gazette
But the words are all too familiar for Cam Howard, 8, of Hampton, who has been diagnosed with the ailment, which is more commonly known as progeria and has been called the rapid aging disease. It's a rare syndrome caused by a mutation in a gene.

Vanda Pharmaceuticals to Participate in the Canaccord Genuity Rare Disease ...
WASHINGTON, Jan. 29, 2015 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) VNDA, -1.74% a biopharmaceutical company focused on the development and commercialization of products for the treatment of central nervous system disorders, today ...

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Scottish Daily Record

Brave five-year-old girl fighting rare disease that's turning cells in her ...
Scottish Daily Record
A MOTHER has told how her little girl is bravely fighting a rare disease that's turning the cells in her body to crystal. Olivia Lamarsh, five, is one of just 2000 people in the world suffering from cystinosis, a terminal illness that stunts growth and ...
Little Olivia's fight against deadly diseaseHerald Scotland

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Therapeutic Focus: Rare Diseases
Medical Marketing and Media
Currently on the cusp of a seismic shift toward innovative, ultra-targeted therapeutics, the rare disease market continues to be dominated by small-molecule medications. Industry analysts have focused their thoughts on a future full of promising gene ...

The Westside Story

Shire deepens rare disease focus with NPS buyout
Royal Society of Chemistry
UK speciality pharmaceutical firm Shire has agreed to buy US biotech NPS Pharmaceuticals for $5.2 billion (£3.5 billion). The move supports Shire's strategy of focusing on treatments for rare diseases, and has been further validated with US approval ...
NPS drug approval bolsters Shire acquisition decisionMedical Marketing and Media
FDA approves NPS Pharma's drug, burnishing Shire's $5.2B betFierceBiotech
NPS Pharmaceuticals, Inc. (NPSP) Wins FDA Approval For Natpara, Validating ...Bidness ETC
Proactive Investors UK -Medical Xpress
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Abiomed Snares Latest Rare-Disease Medtech Approval (ABMD)
Seeking Alpha (registration)
There has been a sudden spate of these rare-disease approvals, as the graph below shows. But Impella RP could treat rather more patients than the 4,000 per year specified by the HDE, so further approvals will be necessary if the RP is to tap its full ...

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Reno Gazette Journal

Letter: Rare Disease Day coming in February
Reno Gazette Journal
I am writing to let fellow Nevadans know about a special event on the horizon. On the last day of February, millions of people around the world will be observing Rare Disease Day. Gov. Sandoval has made a proclamation recognizing Feb. 28, 2015 Rare ...

PBS NewsHour

As Rose Bowl shines spotlight on rare disease, here are three others that ...
PBS NewsHour
David, Lynn, Amy and Mark Frohnmayer are avid University of Oregon fans. David and Lynn founded the Fanconi Anemia Research Fund in 1989 after their three daughters, including Amy, were diagnosed with the rare, life-threatening disease. Photo from ...
Coach discusses rare disease that connects FSU, UOkgw.com
FSU Football Coach Battling Son's Rare Disease Off The FieldFOX 11 Los Angeles
Rose Bowl opponents team up to raise awareness of rare blood disorderNET Website

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