The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…
Spinraza (nusinersen), an injectable medication approved two years ago by the U.S. Food and Drug Administration (FDA) to treat spinal muscular atrophy (SMA), is way overpriced. But so is its biggest potential competitor — Novartis‘ gene therapy Zolgensma — even though both have substantially helped children receiving either…
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…
Visitors to the Clinic for Special Children (CSC) just outside Strasburg, Pennsylvania, might be forgiven for thinking they’ve made a wrong turn. Hidden at the end of a long, winding driveway off Bunker Hill Road, the clinic, which serves the Amish and Mennonite communities of Lancaster County and beyond, looks…
Dozens of films have focused on those with diseases or disabilities — and there’s no shortage of Hollywood productions about love and sex. But only a handful have ever really tried to combine these two themes. “Take a Look at This Heart” does the job with tenderness and finesse.
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
Poland’s Ministry of Health has agreed to pay for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) regardless of age or disease severity — ending a year-long struggle by the country’s nonprofit Fundacja SMA to gain access to one of the world’s most costly drugs. The surprise announcement…
Danyelle Sun, a Milwaukee mother of two children with spinal muscular atrophy (SMA), says Biogen’s Spinraza (nusinersen) has rescued both her kids from potentially lifelong disabilities. Sun, 35, is the Wisconsin state ambassador for NORD, the National Organization for Rare Diseases. Her 7-year-old daughter, Ruby, has SMA type 3,…
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…
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