News

People who undergo surgery to remove the entire thyroid gland have a much higher incidence of chronic hypoparathyrodism than previously reported, with younger patients and those with so-called toxic thyroid disease at particular risk, a study in Denmark found. The study, “High incidence of…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Alizé Pharma 3 is rebranding as Amolyt Pharma and plans to start clinical testing of a potential hypoparathyroidism treatment known as AZP-3601 later this year. “Our new name, Amolyt Pharma, is derived from ammolite, a rare gemstone originating from living organisms,” Thierry Abribat, PhD, the company’s founder and…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

People with hypoparathyroidism living in the U.S. still struggle to receive proper medical care, particularly during emergency room visits, according to findings of a survey conducted by the HypoPARAthyroidism Association (HPA). The results of the HPA’s “Voices of Hypopara” survey were released in support of World Hypoparathyroidism…

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…

A 52-year-old woman with spine and hip pain related to hypoparathyroidism was wrongly diagnosed with spondyloarthritis (SpA) — a type of arthritis that affects the spine — leading to years of unnecessary anti-inflammatory use, a case study reported.  Its…

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…

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.

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…