NORD Opens COVID-19 Financial Aid Program for Rare Disease Community
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community…
Articles by Mary Chapman
Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will…
Clinic Meets Goal Early in SMA Carrier Screening Among Amish, Mennonites
Meeting its goal in less than half the time, the Clinic for Special Children (CSC) in Pennsylvania Dutch Country…
New US Institute for Gene Therapies Seeks to Modernize Regulatory, Reimbursement Framework
The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies…
Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The…
Nonprofits RARE Courage, All Wheels Up Partner on Survey Seeking to Improve Air Travel for Wheelchair Users
Kevin Schaefer hadn’t been in an airport since he was 4 years old, so he had been looking forward to…
Cure SMA Urges States to Start SMA Newborn Screenings This Year
For the new year, Cure SMA is stepping up its efforts to get states not testing for spinal…
Up to 100 Free Zolgensma Treatments Going Out Worldwide in 2020 Under Access Program
Novartis plans to give up to 100 doses annually of Zolgensma, its costly gene therapy for spinal muscular atrophy (SMA),…
Zolgensma Use Strong with About 100 Children Treated Since US Approval, Novartis Says
With about 100 spinal muscular atrophy (SMA) patients already treated with Zolgensma (onasemnogene abeparvovec-xioi) since the gene…
MDA Awards $6.6M to Scientists Working in SMA, Other Neuromuscular Diseases
Some $6.6 million in new Muscular Dystrophy Association (MDA) multiyear grants will support promising studies in an array of…