Larry Luxner,  —

At least three major pharmaceutical companies are looking into potential gene therapies for Huntington’s disease — a rare inherited condition without a cure or a treatment to stop its progression. While several approved therapies exist to control the disorder’s physical and psychiatric effects, non-drug alternatives to alleviate the worst…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases — whose immune systems were generally compromised even before the outbreak — are particularly terrified of this pandemic. That fear is evident in an online survey conducted by the National…

As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases — whose immune systems were generally compromised even before the outbreak — are particularly terrified of this pandemic. That fear is evident in an online survey conducted by the National…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The uncontrolled jerking and writhing movements associated with Huntington’s disease are well known, along with cognitive decline. But doctors who treat Huntington’s patients are far less familiar with the pain and depression their patients endure almost constantly. That’s the consensus of a panel of experts who spoke at…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Enroll-HD, the world’s largest observational study of Huntington’s disease, hopes to add thousands of patients to its registry, reaching a possible total of 30,000 over the next three years. Georg Bernhard Landwehrmeyer, MD, its principal investigator, spoke about the ambitious project at last month’s European Conference on Controversies…