News

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…

Targeting a gene called ALK may prevent weight gain and could lead to effective therapies to combat obesity, a large study in humans and animals suggests. The study, “Identification of ALK in Thinness,” was published in the journal Cell. Genetics play a substantial role in…

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…

Mutations in the gene coding for a protein called adiponectin are associated with a higher likelihood of type 2 diabetes  and obesity. However, this relationship varies with ethnicity, a study in India found. The study, “A genetic analysis identifies a haplotype at adiponectin locus: Association with obesity…

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…

The U.S. Food and Drug Administration (FDA) has accepted and given priority review to Rhythm Pharmaceuticals’ request that setmelanotide be approved to treat people with POMC deficiency and LEPR deficiency, two rare genetic obesity disorders. This priority status is expected to shorten setmelanotide’s regulatory review to…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

Air pollution may be associated with obesity and body fat percentage, according to a comprehensive new study from the United Kingdom. The link between certain air pollutants and obesity may be stronger among individuals who are genetically predisposed to have a higher body mass index (BMI), the findings suggested.

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…