News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

New preclinical data continues to support uniQure’s AMT-130 investigational gene therapy for Huntington’s disease. Results reveal the therapy lowered the levels of toxic huntingtin protein in nerve cells derived from Huntington’s patients as well as in mouse, mini-pig and non-human primate models of the disease. The results were shared…

A potential treatment called RG6042, which target all forms of the mutant huntingtin protein (mHTT) — the underlying cause of Huntington’s disease, has been named an orphan drug by the Ministry of Health,…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

Use of antihypertensives — medications used to treat high blood pressure — is associated with milder disease, slower progression, and delayed clinical onset in people with Huntington’s disease, a study found. The findings suggest an “exciting future avenue to explore the repurposing of specific antihypertensive medications for the…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

Alpha-synuclein, the protein implicated in Parkinson’s disease, may play a role in Huntington’s disease after researchers found elevated levels of the protein in patients, a recent study suggests. The study, “Elevated Serum α-Synuclein Levels in Huntington’s Disease Patients,” was published in Neuroscience.

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…

Linda Lacroix of Milton, Vermont, never imagined that at the age of 68, she’d be working as a lunch lady at the local high school cafeteria. But that’s what she’s had to do to pay the mortgage on the family home so her 67-year-old husband, Joe, has a place to…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…