Larry Luxner,  —

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

Karen Haberberg, a New York portrait photographer, has taken tens of thousands of pictures throughout her career of families, couples, pregnant women, teenagers, and fashion models — not to mention dramatic landscape shots from Iceland to Indonesia. She’s also done several personal photo projects including “Benjamin,” a haunting portrayal…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

Orphan drug development, rising healthcare costs, patient assistance programs and controversial right-to-try legislation are of paramount interest to the parents and caregivers of boys with Duchenne muscular dystrophy (DMD). “One of the chief challenges of 21st Century pediatric medicine is our continued inability to provide more help to children born with…

With nearly 7,000 rare diseases, including Duchenne muscular dystrophy, affecting an estimated 30 million Americans, it’s hard for Paul Melmeyer to keep track of them all. As director of federal policy at the National Organization for Rare Disorders (NORD), Melmeyer’s job is to represent the rare disease patient in…

For years, Lexi Pappas hid the fact she had facioscapulohumeral dystrophy (FSHD) — a hereditary muscle wasting disorder that begins in the face and gradually spreads to the shoulders and upper arms, and sometimes also to the legs. As the disease took its toll on Lexi — causing her…

Thirty-three years ago, doctors told Pat Furlong that her two sons, Christopher and Patrick, had Duchenne muscular dystrophy (DMD). Ten years later, the Ohio nurse — along with other fathers and mothers of Duchenne boys — founded Parent Project Muscular Dystrophy (PPMD) to raise money for Duchenne research and ultimately to…