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Clinical Trials (2)

Improving the PWS Clinical Trial Experience for Families and Clinical Study Site Staff

November 29, 2023

It has been an exciting time for PWS research and clinical trials. The field has been working hard to find effective treatments for the symptoms of PWS to help our children lead more independent and fulfilling lives. In the last

Soleno Therapeutics Announces Positive Statistically Significant Top-line Results from Randomized Withdrawal Period of Study C602 of DCCR for Prader-Willi Syndrome

September 26, 2023

We are excited to share the news that Soleno has announced positive, statistically significant top-line results from the randomized withdrawal period of study c602 of DCCR for Prader-Willi Syndrome. In the study, patients taking DCCR showed a highly significant improvement

Harmony Biosciences Announces Phase 3 Study of Pitolisant

July 20, 2023

Harmony Biosciences has announced plans to begin a Phase 3 study of Pitolisant for individuals with Prader-Willi syndrome ages 6 and up in the 4th quarter of 2023. This announcement is made following a positive end-of-phase 2 meeting with the

Harmony Biosciences Announces Topline Results From Phase 2 Pitolisant Study

November 1, 2022

Today, Harmony Biosciences announced topline data on the primary outcome, excessive daytime sleepiness, from their Phase 2 proof-of-concept trial in patients with Prader-Willi syndrome, or PWS. The study included 65 volunteers, ages 6 to 65, who were randomized 1:1:1 to

Inversago Pharma Receives Rare Pediatric Disease Designation for INV-101 for the Treatment of PWS

January 11, 2021

Inversago Pharma has been granted a Rare Pediatric Disease (RPD) designation by the FDA for the Company’s lead compound, INV-101, for the treatment of Prader-Willi syndrome (PWS). A Phase I study with INV-101 is currently ongoing to evaluate its safety,

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