WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12…
Articles by Larry Luxner
ICER: Spinraza, Zolgensma Both ‘Dramatically Improve’ Lives of Kids With SMA
Spinraza (nusinersen), an injectable medication approved two years ago by the U.S. Food and Drug Administration (FDA) to treat…
Vienna to Host RARE2019 Meeting on Rare Diseases
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the…
Rural Pennsylvania Clinic Treats Amish, Mennonite Children Who Have SMA
Visitors to the Clinic for Special Children (CSC) just outside Strasburg, Pennsylvania, might be forgiven for thinking they’ve made a…
Provocative Film Explores Love and Sexuality Among the Disabled
Dozens of films have focused on those with diseases or disabilities — and there’s no shortage of Hollywood productions about…
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time…
Poland to Reimburse Cost of Spinraza to Treat SMA, Ending Lengthy Struggle
Poland’s Ministry of Health has agreed to pay for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) regardless…
Wisconsin Mom Credits Spinraza with Restoring ‘Quality of Life’ to Her Kids with Type 2 and 3 Disease
Danyelle Sun, a Milwaukee mother of two children with spinal muscular atrophy (SMA), says Biogen’s Spinraza (nusinersen) has rescued…
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range…
Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison…