Rare Disease in the News

NBC Southern California

Following Brother's Death, Irvine Boy to Receive Treatment for Rare ...
NBC Southern California
A fundraiser was held for a boy battling Batten's Disease, after the same disease took his brother's life. The fundraiser will raise money so the family can travel to ...

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The Mighty

Jerika Bolen, Teen With Rare Disease Who Wanted to End Her Own Life, Dies
The Mighty
Jerika Bolen, a 14-year-old girl with spinal muscular atrophy type 2 (SMA), died on Thursday after entering hospice care to end her own life. With medical interventions, SMA – which causes muscle weakness, trouble breathing and feeding difficulties ...
Appleton teen Jerika Bolen, who sparked debate over "right to die", passes awayWGBA-TV
Jerika Bolen dies after ceasing treatmentAppleton Post Crescent
J's Last Dance by Jen Bolen - GoFundMeGoFundMe
WBAY -National Institute of Neurological Disorders and Stroke - National Institutes of Health
all 83 news articles »

CBS News

Unraveling a genetic clue to our "sixth sense"
CBS News
Two young patients with a rare condition have helped scientists unlock a better understanding of our sense of touch and what researchers call our “sixth sense” – our physical awareness of our place in space. The work has identified a gene involved in ...

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Fox News

Nebraska boy who had headache diagnosed with rare disease
Fox News
What his mother first thought was a harmless headache landed 7-year-old Somolon Pyle in the hospital, where he fell into a coma and was subsequently diagnosed with a rare disease. Now, the Omaha, Nebraska, boy is awake and undergoing treatment as ...


12NewsNow.Com

5th grader with rare disease made honorary West Brook cheerleader
12NewsNow.Com
BEAUMONT - The West Brook Bruins are fired up for the Friday Night Lights. At this week's pep rally, many noticed a special guest among the cheerleaders. 5th grader Emily Neild made an appearance. The high school cheerleaders jumped into action to ...


How one rare disease led to painting of blue people
Genetic Literacy Project
The blue people of Troublesome Creek had methemoglobinemia, a metabolic condition affecting hemoglobin, the four-part protein that carries oxygen bound to an iron atom at each subunit's core…it is a tale of an autosomal recessive disease that has ...


USA TODAY

Horizon Pharma buys Raptor for $800 million in rare disease business deal
USA TODAY
The deal, due to close in the fourth quarter, is set to bump up Horizon's portfolio of drugs for rare diseases. Adding Raptor also helps Horizon as it looks to expand to Europe and other markets internationally. Best known for its Procysbi and Quinsair ...
Horizon Pharma in $800M Raptor rare disease buy, but pipeline beset by failuresFierceBiotech
More Rare Disease Dealmaking to Follow Horizon's Raptor DealTheStreet.com
Horizon Buys Rare-Disease Company Raptor for $800 MillionBloomberg
ModernHealthcare.com -PMLiVE -Rare Disease Report
all 153 news articles »

San Francisco Business Times (blog)

BioFlash Podcast: How do you market a drug for a rare disease?
San Francisco Business Times (blog)
Here's what it means to talk about marketing in the context of rare disease drugs. AlexRaths/ThinkStock. Samuel Falsetti was a researcher at the H. Lee Moffitt Cancer Center and Research Institute in Tampa for years, helping discover oncology drugs ...


Forbes

Rare Diseases Are Becoming Too Common. Sound Impossible? Here's Why It's Not
Forbes
It is hard to make money treating rare diseases. There simply aren't enough customers to generate many profits. That's why the U.S. government passed the Orphan Drug Act in 1983, a law which created a series of incentives to encourage drug companies to ...


MacroGenics to Present at the Leerink Partners Rare Disease & Immuno-Oncology Roundtable
GlobeNewswire (press release)
Rockville, MD, Sept. 23, 2016 (GLOBE NEWSWIRE) -- MacroGenics, Inc. (Nasdaq: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, ...

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