Rare Disease in the News


Rare disease could turn boy into 'mannequin'
(NEWSER) – Three-year-old Jarvis Budd is one of just 13 people in Australia to suffer from a rare disease called fibrodysplasia ossificans progressiva, or "human mannequin disease." This means that with every minor fall or injury, he runs the risk of ...
Disease puts 3-year-old boy at risk of becoming 'human mannequin'Fox News

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PLoS Blogs (blog)

Fighting Canavan: Honoring Rare Disease Week
PLoS Blogs (blog)
“Trying to explain to someone what it's like to raise, love, and save a child born with a rare disease is difficult, and painful beyond words. The world of rare diseases can be dark, depressing and lonely. To raise awareness about a specific rare ...

Girl with rare disease finds a best friend in her service dog
My Fox Boston
WOBURN, Mass. (MyFoxBoston.com) -- This week, millions of Americans are recognizing Rare Disease Day, and one local family wants the world to know about a rare disease affecting their 10-year-old daughter, and a huge new helper by her side.


FDA Recognizes Rare Disease Day 2015
Rare Disease Day, which is commemorated internationally on the last day in February, was established to raise awareness about rare diseases, the challenges encountered by those affected, the importance of research to develop diagnostics and treatments, ...
BI rare-disease awareness push slated for SaturdayMedical Marketing and Media
Health Commissioner calls for national plans to tackle rare diseasesEurActiv
The Importance of Rare Disease EducationMedscape
Asbestos.com (blog) -580 CFRA Radio
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Boston Business Journal (blog)

With the help of patient groups, biotechs hone in on rare diseases
Boston Business Journal (blog)
Bob Coughlin, president of MassBio (left) with speakers at an event held this week for Rare Disease Day: Megan Duff, a neurofibromatosis type 1 patient; Lisa Cimino, mom of a focal segmental glomerulosclerosis patient; Ilene Sussman, executive director ...

Daily News & Analysis

Steve Waugh Foundation lends support to rare disease sufferers
Daily News & Analysis
Waugh added that medicines are often 'very expensive', adding that there is 'very little respite care' for parents, and siblings in a lot of ways 'get left behind a bit' because when one has a child with a rare disease they are so focused on getting ...
Steve Waugh raises rare disease awarenessNEWS.com.au

all 14 news articles »


Boy's rare disease triggers push for newborn screening
A-L-D or Adrenoleukodrystrophy is a rare progressive neurological disorder. Child Neurologist Dr. Chris Miller sees Blake in Knoxville. "The patient is robbed increasingly of their ability to use their muscles and eventually to use their senses like ...

Pursuing Elusive Diagnoses for Rare Diseases
Editor's Note: There are no published statistics on the number of undiagnosed "rare disease" patients currently in the United States. The accepted definition of a rare disease is one that affects fewer than 200,000 Americans at any given time ...

Cost concerns at center of debate over rare disease treatment
Nashoba Publishing
Boston Biomedical Associates Vice President of Operations Ken Dhimitri said while about 30 percent of all new drug applications are for rare diseases and approval rates are "about even" between rare and common diseases, 95 percent of rare diseases lack ...

4-month-old girl fights rare 'maple syrup urine' disease
The girl's mother, Stacie Maynard, told FOX59, “I want people to realize that this disease is out there. It is rare, but it is out there. To raise awareness of the newborn screening and how helpful it is to be diagnosed early on.” Rare Disease Day ...