Treatment of rare diseases

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Soleno Therapeutics Announces Presentations Featuring VYKAT(TM) XR in Prader-Willi Syndrome at PES 2025
Globenewswire· 2025-05-15 12:00
Core Insights - Soleno Therapeutics, Inc. announced poster presentations for its VYKAT XR clinical development program at the Pediatric Endocrine Society Meeting 2025, focusing on hyperphagia treatment in Prader-Willi syndrome (PWS) [1][2] Group 1: Company Overview - Soleno Therapeutics is a biopharmaceutical company dedicated to developing novel therapeutics for rare diseases [10] - The company's first commercial product, VYKAT XR, is a once-daily oral treatment for hyperphagia in individuals aged 4 years and older with PWS [10] Group 2: Product Information - VYKAT XR (diazoxide choline) was approved by the U.S. FDA on March 26, 2025, and is now available for U.S. patients [4] - The product is indicated for treating hyperphagia associated with PWS [4] Group 3: Clinical Research - Presentations at PES 2025 include studies on long-term administration of VYKAT XR and its effects on lean body mass and behavioral symptoms in PWS [2][3] - The studies highlight significant improvements in hyperphagia and behavioral symptoms upon resuming treatment after a randomized withdrawal period [2]
Soleno Therapeutics Announces Oral Presentations Featuring VYKAT(TM) XR in Prader-Willi Syndrome at ESPE and ESE 2025
Globenewswire· 2025-05-08 20:05
Core Insights - Soleno Therapeutics, Inc. announced the presentation of data from its VYKAT XR clinical development program for treating hyperphagia associated with Prader-Willi syndrome (PWS) at the upcoming Joint Congress of ESPE and ESE Meeting 2025 [1][2] Company Overview - Soleno Therapeutics is focused on developing and commercializing novel therapeutics for rare diseases, with its first commercial product being VYKAT XR, an oral treatment for hyperphagia in patients aged 4 years and older with PWS [10] Product Information - VYKAT XR (diazoxide choline) was approved by the FDA on March 26, 2025, and is now available to U.S. patients [2] - The product is indicated for the treatment of hyperphagia in adults and pediatric patients with PWS [4] Clinical Presentation Details - The company will present a study titled "The Impact of Prader-Willi Syndrome (PWS) on Caregivers and the Healthcare System: A Burden of Illness Study Design" in a poster format on May 11 and May 12, 2025 [2] - An oral presentation titled "Developmental Behaviour Checklist 2 Response and Relationship to Hyperphagia Reductions" is scheduled for May 13, 2025 [6] Understanding Prader-Willi Syndrome - PWS occurs in approximately 1 in every 15,000 live births, characterized by hyperphagia, which can severely impact the quality of life for affected individuals and their families [3] - Additional symptoms include behavioral problems, cognitive disabilities, low muscle tone, and increased risk of co-morbidities such as diabetes and cardiovascular disease [3]
Quoin Pharmaceuticals Announces Additional Positive 'Whole Body' Clinical Data from Ongoing Pediatric Netherton Syndrome Study and Approval to Initiate Testing of a Second Pediatric Patient
Newsfilter· 2025-04-02 11:30
Core Insights - Quoin Pharmaceuticals Ltd. has reported highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome study, indicating that after 6 weeks of treatment with QRX003, the patient's skin is almost completely healed and there is a significant reduction in the need for other medications [1][2][6] - The treatment has led to the elimination of pruritus, allowing the patient to experience uninterrupted sleep for the first time, which is a critical improvement in quality of life for those suffering from Netherton Syndrome [2][6] - No adverse events have been reported during the 6-week treatment period, and the company has received approval to initiate treatment on a second pediatric patient [2][6] Company Overview - Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products for rare and orphan diseases [5] - The company is committed to addressing unmet medical needs and has a pipeline that includes four products targeting various rare conditions, including Netherton Syndrome [5] Product Information - QRX003 is a topical lotion designed to treat Netherton Syndrome by promoting a normalized skin-shedding process and strengthening the skin barrier [4] - The product utilizes a proprietary delivery technology and contains a broad-spectrum serine protease inhibitor, which mimics the function of the LEKTI protein absent in patients with Netherton Syndrome [4]