MDA Hellas Gives Hope to SMA, Duchenne Patients in Greece
Either by coincidence or by design, MDA Hellas, the Muscular Dystrophy Association of Greece, is located on Elpidos Street…
Articles by Larry Luxner
Canada Trails US in Patient Access to Rare Disease Therapies, CORD President Says
Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing…
US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn…
#ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says
Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential…
#ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a…
#CureSMA2018 – Science and Families at Center of Next Month’s Cure SMA Conference
Cure SMA will host the world’s largest regular gathering on spinal muscular atrophy next month in Dallas, bringing together leading SMA…
#ERDC2018 – New EU Law to Protect Patient Data Could Affect Rare Disease Registries
Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough…
Australia Adds Spinraza to Subsidized Coverage for SMA Kids, But Malaysia Still Waiting
If 8-year-old Branden Lim had been born in Australia instead of Malaysia, his parents soon would have no problem obtaining…
‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and…
#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in…