Posts by: Larry Luxner

News

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 diagnosed. ... 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 than ... Read more

April 4, 2019April 4, 2019

At Vienna Conference, Gaucher Experts Discuss Treatment of ‘Model Disease’

News

Gaucher disease took center stage at a recent medical conference in Vienna, Austria, organized by Ari Zimran, MD, director of the Gaucher Clinic at Israel’s Shaare Zedek Medical Center. “Gaucher ... Read more

March 21, 2019March 21, 2019

Britain’s Madeline Collin Strives to be Voice of Hope for Other Gaucher Disease Patients

News

Discouraged by doctors. Ridiculed by classmates. Challenged on the homefront. Despite the physical and emotional trauma endured by Madeline Collin — who has Gaucher disease — this cheerful 24-year-old says her ... Read more

March 14, 2019

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

News

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 this ... Read more

March 11, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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 to ... Read more

February 26, 2019February 25, 2019

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

News

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 already ... Read more

February 20, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

News

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 March ... Read more

February 5, 2019February 5, 2019

ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration

News

G71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, and G40.419 means Dravet syndrome. All three designations became official on Oct. 1, 2018, joining some ... Read more

December 21, 2018December 21, 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat ... Read more