Posts by: Larry Luxner

News

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials ... Read more

May 27, 2019May 27, 2019

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

cystic fibrosis

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site ... Read more

May 17, 2019May 17, 2019

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

cystic fibrosis

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been ... Read more

May 16, 2019May 16, 2019

Bike Rides, Walkathons, Other Fundraising Events Mark CF Awareness Month

cystic fibrosis, News

When it comes to disease “awareness” days, weeks, and months, 2019 is crammed with hundreds of them. May leads the calendar with 37 such designations, calling attention to everything from ... Read more

May 1, 2019May 1, 2019

PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights

News

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for ... Read more

April 25, 2019April 25, 2019

Nonprofit Group Works to Raise Rare Disease Awareness in India

News

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients ... Read more

March 14, 2019April 4, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

cystic fibrosis

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of ... Read more

March 11, 2019March 26, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

cystic fibrosis, News

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

February 25, 2019February 25, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

cystic fibrosis

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have ... Read more

February 20, 2019March 5, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

cystic fibrosis

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The ... Read more

Posts by: Larry Luxner

Larry Luxner

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

Bike Rides, Walkathons, Other Fundraising Events Mark CF Awareness Month

PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights

Nonprofit Group Works to Raise Rare Disease Awareness in India

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

Vienna to Host RARE2019 Meeting on Rare Diseases

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