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Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Growth hormone therapy should be universally available, given to adults as well as children with Prader–Willi syndrome (PWS) and based solely on a genetic PWS diagnosis. This is the position of the Clinical and Scientific Advisory Board of the…
Tonix Pharmaceuticals has licensed technology from the French National Institute of Health and Medical Research (Inserm) that it aims to use for treating failure to thrive in Prader-Willi syndrome. The license will allow Tonix to expand its intranasal — administered via the nose — potentiated oxytocin program to…
Tryp Therapeutics announced plans to launch a Phase 2a clinical trial evaluating the potential of its investigational oral candidate TRP-8802 (synthetic psilocybin) at reducing excessive appetite in people with Prader-Willi syndrome (PWS) and other rare disorders characterized by overeating and with high unmet therapeutic needs. The upcoming trial is…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. 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…
The COVID-19 pandemic — which took a heavy toll on people with Prader-Willi syndrome (PWS) and their families, according to a recent webinar — likely influenced the results of the DESTINY PWS Phase 3 trial that was testing the investigational therapy diazoxide choline controlled release (DCCR) at its…
The antidepressant sertraline may help control temper outbursts in people with Prader-Willi syndrome (PWS), a small study suggests. Among 13 patients who completed six months of treatment with sertraline, the researchers reported a greater than 90% decrease in the frequency of temper outbursts. Treatment over six months also…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
As children with Prader-Willi syndrome (PWS) age, gains in weight are among concerns that considerably affect both their quality of life and that of family members, with rising stress and levels of caregiver burden evident, a survey of small group of parents reports. The study based on parent responses,…
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