Posts by: Larry Luxner

News

What began in the early 1990s as an informal gathering of seven European patient groups in the Italian port of Trieste has evolved into the world’s largest charity for Gaucher ... Read more

September 26, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of ... Read more

August 29, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues ... Read more

August 20, 2019August 19, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve ... Read more

August 5, 2019August 5, 2019

Psychologist Living with Gaucher Strives to Be ‘Role Model of Success’

News

When school psychologist Wayne Rosenfield was 5 years old, he had an enlarged spleen. Doctors in his hometown of Springfield, Massachusetts, brushed it off, telling his parents: “He’s just a ... Read more

August 2, 2019August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, ... Read more

July 25, 2019July 25, 2019

Centogene Rolls Out Free Biomarker App for Gaucher Patients

News

Germany’s Centogene is introducing a free smartphone app that lets people with Gaucher disease monitor their own progress. But an organization that represents Gaucher patients is concerned about what the ... Read more

July 18, 2019

European Initiative Targets Diagnosis, Treatment of Rare Diseases

News

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments ... Read more

June 25, 2019

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

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 of ... Read more

May 27, 2019May 24, 2019

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

News

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 was ... Read more