Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have.
Articles by Larry Luxner
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second…
European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis…
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet…
Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups…
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails…
Nonprofit Group Works to Raise Rare Disease Awareness in India
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country.
MDA Conference Bringing Neuromuscular Disease Experts to Orlando to Share ‘New Era’ in Treatment, Research
For more than four decades, comedian Jerry Lewis hosted the MDA Labor Day Telethon on behalf of the Muscular…
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves…
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and…