Posts by: Larry Luxner

Larry Luxner

June 4, 2018June 4, 2018

US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third ... Read more

May 29, 2018May 29, 2018

#ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 17, 2018May 17, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

May 2, 2018May 2, 2018

#ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan ... Read more

April 13, 2018April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

News

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

March 19, 2018March 19, 2018

Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

News

Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare ... Read more

March 14, 2018

‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event

News

When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD,  for short. Hunter Pageau, a 12-year-old boy from ... Read more

March 12, 2018

Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

News

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new ... Read more

March 9, 2018

US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs

News

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated ... Read more