Rare Disease in the News

DOH aims to make tuberculosis a rare disease by 2022 | Inquirer ...
Inquirer.net
The Department of Health is shifting its anti-tuberculosis campaign to make tuberculosis a rare disease instead of a prevalent illness by 2022. According t.

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'Terrifying and isolating' - living with a rare disease
BBC News
"It's terrifying, isolating - you're having these symptoms and you don't know what tomorrow is going to bring or where it will end up." That is how Carol McCullough describes living with a rare disease and trying to find out what was wrong with her.


MyDaytonDailyNews

2-year-old with rare disease becomes honorary police officer in NJ
MyDaytonDailyNews
A 2-year-old with a rare genetic disease became the youngest police officer in New Jersey, WABC reported. >> Read more trending news. Trent Powers is battling Duchenne muscular dystrophy, and on Wednesday he became an honorary member of the ...
2-year-old with rare disease becomes honorary police officer in NJ ...WSB Radio
2-year-old with Duchenne muscular dystrophy made honorary Spotswood police officerNews 12 New Jersey

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Endpoints News

Ultragenyx shares tumble lower after rare disease drug fails PhII trial
Endpoints News
Shares of Ultragenyx $RARE tumbled close to 10% after the rare disease biotech reported that one of its drugs failed a Phase II trial. UX007 was tested as a treatment for glucose transporter type-1 deficiency syndrome—or Glut1 DS—among a small group ...
Ultragenyx Looks Ahead After Triheptanoin FailureSeeking Alpha
SEC FORM 4SEC

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Refinery29

The Amazing Reason This Girl Flew Halfway Across The Country To Meet A Stranger
Refinery29
Hypopara, or hypoparathyroidism, is a rare condition that causes someone's body to produce a low amount of a hormone called parathyroid hormone, according to Hypopara UK. Having too little of this hormone causes insufficient levels of calcium in the ...


Northern Pen

Rare disease encourages Chris Humby to not quit, stay involved
Northern Pen
This past October, Humby began a treatment that is supposed to help stop, and maybe even reverse, the effects of the disease. For two days he gets intravenous immunoglobulin (IVIg) therapy. It contains plasma from human blood and helps boost a person's ...


Erytech investigates possible rare disease therapy
The Pharma Letter (registration)
Erytech Pharma (Euronext: ERYP), a French biopharmaceutical company, will collaborate with the Fox Chase Cancer Center in Philadelphia, USA, to develop the company's rare disease candidate erymethionase. Erytech is developing the compound as a ...


Health Affairs (blog)

For Rare Disease Patients, A Pathway To Hundreds Of New Therapies
Health Affairs (blog)
Many rare disease patients use drugs off-label based on limited data because they have no better options available. In fact, it is estimated that 95 percent of rare diseases do not yet have a single FDA-approved treatment. One of the authors of this ...


Isanti County News

Former Cambridge business owner diagnosed with rare disease
Isanti County News
But in March 2015, Southerland was forced to close his business after being diagnosed with a rare disease, paraneoplastic syndrome, which is triggered by an altered immune system response to abnormal tissue growth in the body and for which there are no ...


Undark Magazine

A Rare Disease? Or No Disease at All?
Undark Magazine
That clearly happens,” said Dr. Stanford Shulman, an infectious disease specialist at Lurie Children's Hospital and Northwestern University, in Chicago. “What's unclear is whether there are any specific infectious triggers that are responsible for that ...