Mary Chapman, features writer —

As the 118th U.S. Congress begins its work, the National Organization for Rare Disorders (NORD) is showcasing the importance of engaging in health policy and what rare disease patient advocates can do to make their voices heard. The organization is the leading patient advocacy organization dedicated to improving the…

The national outcomes-based patient support organization Curant Health has joined rare360, a Rare Advocacy Movement (RAM) partnership program that seeks to connect, build up, and protect community-focused industry, academic, and advocacy stakeholders in rare disorders. “Being a partner with the Rare Advocacy Movement (RAM) and a registered…

Medable, a technology platform for clinical trials, has partnered with the nonprofit Every Cure to speed the discovery of treatments for rare diseases — such as aromatic l-amino acid decarboxylase (AADC) deficiency — with the help of drug repurposing. The partners will focus on identifying new uses…

In marking the 40th year of the Orphan Drug Act (ODA) — used by the U.S. Food and Drug Administration (FDA) to spur treatment development for rare diseases — a life sciences industry coalition is calling on legislators and other policymakers to make a new commitment to finding therapies…

As U.S. lawmakers prepare to head home for the holidays, the National Organization for Rare Disorders (NORD) held out hope they would still act to ensure that people with rare diseases such as aromatic l-amino acid decarboxylase (AADC) deficiency get timely access to treatments and diagnostic tools. On…

Since its founding more than 25 years ago, Eurordis has presented and promoted initiatives to ensure that people with rare diseases, such as aromatic l-amino acid decarboxylase (AADC) deficiency, are at the heart of treatment development. That work continues — and indeed, Eurordis is stepping up its…

An upcoming U.S. survey, reported to be the first of its kind, aims for a better understanding of barriers to healthcare access and affordability among rare disease patients and caregivers in racial minority or other marginalized groups, and to help inform ways to remove them. The initiative is led by…

While the National Organization for Rare Disorders (NORD) is pleased Congress voted to reauthorize the Food and Drug Administration’s (FDA) user fee program — as part of a U.S. government short-term spending package (HR 6833) — it noted the legislation falls short in areas important to…

A free live webinar on Sept. 20 will explore various aspects of rare disease clinical trials — and offer insight into the challenges researchers face in finding and enrolling participants, particularly those of diverse backgrounds. Those interested in attending can register now for the hour-long webinar, titled “Rare Disease…

The NORD Rare Diseases & Orphan Products Breakthrough Summit, set for Oct. 17–18, will feature a range of speakers and discussions about issues affecting the more than 300 million people worldwide thought to have a rare disease — including those with aromatic l-amino acid decarboxylase (AADC) deficiency. The…