News

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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 ... Read more

June 16, 2020June 16, 2020

Saniona Planning Phase 2b Trial of Tesomet in Controlling Hunger

News

The U.S. Food and Drug Administration (FDA) gave positive feedback to Saniona regarding its work to advance Tesomet as a possible treatment to aid weight loss in people with ... Read more

June 11, 2020June 11, 2020

DCCR Fails to Reduce Excessive Appetite But Improves Other Measures, Data Show

News

Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled-release (DCCR) failed to significantly lower excessive appetite, but it did improve body composition and behavior in Prader-Willi syndrome (PWS) patients, top-line ... Read more

June 9, 2020June 9, 2020

Lack of Vitamin D in Diet Linked to Lower Levels in PWS Patients in Study

News

Vitamin D levels are lower among adults with Prader-Willi syndrome (PWS) that in other obese individuals, mainly because PWS patients tend to consume less vitamin D through their diet, ... Read more

June 9, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

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 ... Read more

June 4, 2020June 4, 2020

Tracking Eye Movements Can Measure Chronic Desire to Overeat

News

Tracking the eye movements of children with Prader-Willi syndrome (PWS) may be a non-invasive, low-cost, and reliable method to assess their chronic desire to overeat, known as hyperphagia, a ... Read more

June 2, 2020June 2, 2020

Liraglutide Helped Maintain Rapid Weight Loss in Teenager With PWS

News

Severe fluid and calorie restriction induced rapid weight loss that was effectively maintained with liraglutide, according to a case report of a young woman with Prader-Willi syndrome (PWS). Titled “Successful rapid ... Read more

May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

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 ... Read more

May 26, 2020May 26, 2020

Researchers Find Corticol Deficiency Rare in PWS, Advise Against Routine Hydrocortisone Use

News

Central adrenal insufficiency, a deficiency in cortisol levels due to problems in the pituitary or hypothalamus, is rare in people with Prader-Willi syndrome (PWS), affecting 1.2% of patients, a study ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

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 ... Read more

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