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The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
Growth hormone therapy, a common treatment for children with Prader-Willi syndrome (PWS),  is also safe and effective at increasing lean muscle mass and lowering body fat in adults with this disease, a review study reports. More work is needed, however, to confirm the therapy’s effects on body composition, as well…
The case of a woman with Prader-Willi syndrome (PWS) who developed slowly progressive insulin-dependent diabetes mellitus, or SPIDDM — usually type 2 diabetes in adults — underscores the need to test patients for disease-causing antibodies and genetic markers, a Japanese…
More than 70% of children and adolescents with Prader-Willi syndrome (PWS) have a high risk of developing autism spectrum disorder (ASD), according to a study in Chinese patients. The findings also indicated that individuals who were shorter, had excess weight, and experienced obstructive sleep apnea — a condition in…
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…
Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare Diseases to heighten awareness about rare disease communities and salute the creativity of its members. Awardees will be able to display their art and…
Due to the COVID-19 pandemic, the Prader-Willi Syndrome Association (PWSA) in the U.S. has announced its upcoming 36th National Convention,  “Hope’s on the Horizon,” will be moving to an all-new virtual format. The PWSA will provide patients and families within the…
Intranasal use of the hormone oxytocin for three months had positive effects on social and eating behavior in boys with Prader-Willi Syndrome (PWS), according to a small clinical trial. The results suggest intranasal oxytocin also may be an…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
Inversago Pharma‘s INV-101 has been granted rare pediatric disease (RPD) designation by the U.S. Food and Drug Administration for the treatment of Prader-Willi syndrome (PWS). This designation is given to potential medications targeting serious diseases that primarily affect less than 200,000 Americans ages 18 or younger. If the…
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