Posts by: Larry Luxner

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

March 2, 2018

Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

News

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in ... Read more

February 26, 2018

BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

News

In recognition of Rare Disease Day 2018, BioNews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies ... Read more

Posts by: Larry Luxner

Larry Luxner

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

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

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

NIH Agency Pioneers Collaborative Research into Rare Diseases

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

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

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

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

Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

Pin It on Pinterest