Posts by: Larry Luxner

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 diseases ... Read more

March 15, 2018March 15, 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 North ... Read more

March 12, 2018March 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 therapies ... Read more

March 9, 2018March 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 30 ... Read more

March 2, 2018March 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 Washington, ... Read more

February 27, 2018February 27, 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 and ... Read more

February 20, 2018February 20, 2018

Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

News

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, ... Read more

February 12, 2018March 15, 2018

Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

News

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine ... Read more

January 31, 2018January 30, 2018

NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

News

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 ... Read more

November 21, 2017November 21, 2017

Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

News

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part ... Read more