Larry Luxner,  —

This month’s Democratic takeover of the U.S. House of Representatives following the Nov. 6 midterm elections could be good news for Americans with rare diseases, says the National Organization for Rare Disorders (NORD), which represents 280 patient advocacy organizations. “The entire community advocated against the repeal of the Affordable Care…

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 Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

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…

One of New England’s top experts in Duchenne muscular dystrophy will give the keynote address at a conference next month dedicated to finding a cure for the neuromuscular disease. Brenda Wong, MD, is the director of the Duchenne program at University of Massachusetts Medical in Worcester, about a 45-minute…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

For both children and adults with Duchenne or Becker muscular dystrophy, having a specially trained dog doesn’t just relieve their loneliness. These service dogs also help patients in dozens of practical ways, doing everything from taking off shoes to picking up toys that fall on the floor. Tali…

Caring for a son with Duchenne muscular dystrophy is hard enough. Imagine how much harder it is when you’ve lost your other son to the same disease. That’s the uncertainty DeAnne Friar of Grand Rapids, Michigan, lives with day after day. Her first child, 20-year-old Colleen, attends Western Michigan University…

Two small patient advocacy groups, Little Hercules Foundation and Team Joseph, are stepping up their joint Duchenne Family Assistance Program (DFAP). The expanded program, created last October, comes in response to the increasing rate of insurance company denials of life-extending treatments and medical devices for Duchenne muscular dystrophy patients.

Duchenne muscular dystrophy affects an estimated 1 in 3,500 male births. But Elizabeth Heller is an outlier even among her rare friends. The Chicago-born Heller, who relocated to Tempe, Arizona, three years ago, is one of only a handful of women with Duchenne in the United States. According to the…

The nonprofit group CureDuchenne and Connecticut-based rareLife Solutions have launched DuchenneXchange — a dedicated online platform where those interested in Duchenne muscular dystrophy “can share comprehensive, safe, credible resources and information, as well as get the support they need from diagnosis to treatment to clinical trials.” The project, announced in a …