Vanderbilt clinical trial offers hope for families battling rare genetic disorder

Vanderbilt University Medical Center is recruiting participants for the Phase 3 Hunger Elimination or Reduction Objective ...

Rhythm Pharmaceuticals Rises on Positive Data for Prader-Willi Syndrome Treatment

The Boston-based pharmaceutical company said the Phase 2 trial showed that setmelanotide was effective in reducing body mass index and hyperphagia in patients with Prader-Willi syndrome, a genetic ...
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Rhythm Pharma hits record high on unexpected obesity-tied win

Rhythm Pharmaceuticals said Thursday said it would send it would send its leading drug into registrational testing for Prader ...
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Acadia starts Phase 3 study for Prader-Willi hunger treatment

Acadia Pharmaceuticals (NASDAQ:ACAD) has initiated a Phase 3 study for carbetocin nasal spray, also known as ACP-101, in the treatment of hyperphagia in patients with the rare genetic disorder ...
Monthly Prescribing Reference

Intranasal Carbetocin Misses Endpoints in Prader-Willi Syndrome Trial

Treatment with intranasal carbetocin was not associated with a statistically significant improvement in hyperphagia compared with placebo. Topline results were announced from a phase 3 trial ...
The American Journal of Managed Care

Growth Hormone Treatment May Improve Sleep Efficiency in Prader-Willi Syndrome

With sleep apnea and abnormal sleep patterns typifying Prader-Willi syndrome, researchers from Sweden and Denmark theorized that treatment with growth hormone may improve both for individuals affected ...
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US FDA approves first treatment for rare genetic disorder Prader-Willi syndrome

(Reuters) -The U.S. Food and Drug Administration on Wednesday approved Soleno Therapeutics' drug to treat a rare genetic disorder, making it the first treatment available for patients who experience ...
Nasdaq

Tonix Pharmaceuticals Provides Overview of TNX-2900 Program for the Treatment of Prader-Willi Syndrome at the Foundation for Prader-Willi Research Family Conference

CHATHAM, N.J., Oct. 09, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced that ...
Monthly Prescribing Reference

FDA Extends Review of DCCR for Prader-Willi Syndrome

A new PDUFA target action date of March 27, 2025 has been set for the application. The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for ...
Business Wire

Acadia Pharmaceuticals Initiates Pivotal Phase 3 Study of Carbetocin (ACP-101) for the Treatment of Hyperphagia in Prader-Willi Syndrome

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the initiation of the Phase 3 COMPASS PWS study evaluating the efficacy and safety of carbetocin nasal spray (ACP ...
Yahoo

A rare disease that causes insatiable hunger

Affected populations: This genetic, multisystem disorder affects an estimated 1 in 30,000 to 1 in 10,000 people worldwide. Most cases of the syndrome occur sporadically, meaning the genetic changes ...
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FDA rejects new drug application for internasal carbetocin to treat Prader-Willi syndrome

Please provide your email address to receive an email when new articles are posted on . The FDA’s Division of Psychiatry has rejected a new drug application for treating hyperphagia, anxiousness and ...