With Zolgensma’s Approval, Debate Shifts to Pricing and Availability of World’s Costliest Drug
Zolgensma, approved for children with spinal muscular atrophy (SMA) up to 2 years old, has two claims to…
Articles by Larry Luxner
Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups…
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails…
PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for…
Nonprofit Group Works to Raise Rare Disease Awareness in India
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country.
MDA Conference Bringing Neuromuscular Disease Experts to Orlando to Share ‘New Era’ in Treatment, Research
For more than four decades, comedian Jerry Lewis hosted the MDA Labor Day Telethon on behalf of the Muscular…
SMA Newborn Screening Expands as More States Enact Mandatory Testing
Five states — Missouri, Minnesota, New York, Pennsylvania and Utah — now routinely screen newborn babies for spinal muscular atrophy…
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves…
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and…
In UK and Ireland, SMA Patients Demand Reimbursement for Spinraza
A drug appraisal committee of Britain’s National Institute for Health and Care Excellence (NICE) met in Manchester, England, for…