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EU Panel Rejects Acadia Pharmaceuticals Rett Syndrome Drug
03 Feb 2026 17:58 GMT
… (trofinetide) for Rett syndrome in adult and pediatric … approved for treating Rett syndrome, a rare genetic … skills and language. Rett syndrome primarily affects females.
In … patients with hyperphagia in Prader-Willi syndrome (PWS).
Intranasal carbetocin did …
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Investigation reveals surge in Georgia special education complaints
04 Feb 2026 18:30 GMT
… cognitive gene disorder - atypical prader willi syndrome - that affects her ability …
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Withdrawal from oral therapy tied to worsened hyperphagia in Prader-Willi syndrome
09 Jan 2026 15:57 GMT
… ; Metabolism, people with Prader-Willi syndrome who previously received diazoxide choline … choline extended release in Prader-Willi syndrome, which all showed … metabolic laboratory values and Prader-Willi syndrome-associated typical behavior problems.” …
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The untold story of life with Prader-Willi syndrome - according to the siblings who live it
07 Jan 2026 03:12 GMT
… sisters of people with Prader-Willi syndrome.
Prader-Willi syndrome is a rare genetic … siblings of individuals with Prader-Willi Syndrome: An integrative systematic review … siblings of individuals with Prader-Willi Syndrome: An integrative systematic review …
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Siblings of people with Prader-Willi syndrome face hidden emotional burdens
07 Jan 2026 01:20 GMT
… and sisters of people with Prader-Willi syndrome.
Prader-Willi syndrome is a rare genetic … family, including the person with Prader-Willi syndrome," she added.
This … of siblings of individuals with Prader-Willi Syndrome: An integrative systematic review…
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Rhythm advances lead asset into late-stage trial in Prader-Willi syndrome
11 Dec 2025 21:18 GMT
… a rare genetic disorder called Prader-Willi syndrome following a promising mid-stage …
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Rhythm Pharmaceuticals Announces Preliminary Data from Exploratory Phase 2 Trial that showed Setmelanotide Demonstrated Positive Efficacy Signal in Prader-Willi Syndrome
11 Dec 2025 15:51 GMT
… of setmelanotide in patients with Prader-Willi syndrome (PWS). The Company also … setmelanotide’s established profile. About Prader-Willi Syndrome
PWS is a rare genetic … obesity associated with other genetic syndromes and general (polygenic) obesity.
…
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Rhythm Pharmaceuticals Announces Preliminary Data from Exploratory Phase 2 Trial that showed Setmelanotide Demonstrated Positive Efficacy Signal in Prader-Willi Syndrome
11 Dec 2025 12:30 GMT
… of setmelanotide in patients with Prader-Willi syndrome (PWS). The Company also … setmelanotide’s established profile. About Prader-Willi Syndrome
PWS is a rare genetic … obesity associated with other genetic syndromes and general (polygenic) obesity.
…
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Rhythm Pharma To Present Preliminary Phase 2 Data Of Setmelanotide In Prader-Willi Syndrome Today
11 Dec 2025 08:58 GMT
… with Prader-Willi syndrome during a live webcast scheduled for December 11, 2025.
Prader-Willi syndrome … hunger associated with Bardet-Biedl syndrome or biallelic POMC, PCSK1, or …
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Rhythm Pharmaceuticals to Announce Preliminary Data from Exploratory Phase 2 Trial Evaluating Setmelanotide in Prader-Willi Syndrome
10 Dec 2025 21:01 GMT
… of setmelanotide in patients with Prader-Willi syndrome (PWS). Conference Call Information … obesity associated with other genetic syndromes and general (polygenic) obesity.
… fatal adverse reactions including “gasping syndrome” can occur in neonates and …
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