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Rare Disease Day 2026: Why Innovation in Rare CNS Disorders Matters More Than Ever
Every year on Rare Disease Day, the world pauses to remember what lies behind the numbers. More than 300 million people worldwide are living with a rare disease, but the real story is the parent sitting in another waiting room with no answers, the child who aged out of a pediatric program before a treatment was found, the family reordering their entire life around a condition that most doctors have never seen. These are the people at the center of everything we do. For those
Feb 283 min read


A New Milestone in Prader-Willi Syndrome Care: FDA Approves VYKAT™ XR (DCCR) for Hyperphagia
Prader-Willi syndrome (PWS) is a rare genetic disorder that disrupts multiple aspects of life, from an insatiable appetite (hyperphagia)...
Apr 17, 20252 min read


Advancing Treatments for Prader-Willi Syndrome: Key Insights from the Miller 2025 Review
An overview of a recent review by Miller et al. (2025) exploring current and emerging therapies for Prader-Willi Syndrome (PWS)
Mar 10, 20252 min read


Raising Awareness for PWS Awareness Month: Join the Effort!
May is PWS Awareness Month, a time dedicated to increasing understanding of PWS and supporting the quest for treatments and a cure.
May 16, 20242 min read
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