Daybue (trofinetide)
Research Synopsis
- Daybue (trofinetide) is the first FDA-approved medication specifically for treating Rett syndrome, a rare neurodevelopmental disorder predominantly affecting females.
- The drug received approval in March 2023 and is intended for use in patients aged 2 years and older, signifying a major breakthrough for an unmet medical need.
- Trofinetide is derived from a neuroprotective peptide that enhances neuronal structure and function, although its precise mechanism of action remains unclear.
- Clinical studies have demonstrated that trofinetide significantly improves behavioral scores and communication abilities in affected individuals, making it a promising treatment option.
- Research has shown that the drug has a favorable safety profile, with no significant drug interactions noted and primarily mild side effects, such as gastrointestinal issues like diarrhea and vomiting.
- Ongoing studies are vital to explore the long-term effects and risks associated with trofinetide treatment, particularly concerning gastrointestinal comorbidities.
- The approval of trofinetide opens avenues for further research on its application in related disorders, such as Fragile X syndrome and autism spectrum disorder.
- Analysis of multiple clinical trials indicates that trofinetide enhances caregiver-reported clinical outcomes, particularly in behavioral assessments.
- The development of trofinetide exemplifies effective collaboration among researchers and pharmaceutical companies, emphasizing the importance of addressing rare diseases through innovation and targeted therapy.
Daybue (trofinetide)
Trofinetide: First Approval.
London, UK
2 hours ago
1 Received
- Trofinetide (DAYBUE™) is a synthetic oral medication designed to treat rare childhood neurodevelopmental disorders, specifically Rett syndrome.
- It is based on a modified version of a tripeptide related to insulin-like growth factor-1 (IGF-1) and is developed by Neuren Pharmaceuticals and Acadia Pharmaceuticals.
- The drug received approval in March 2023 in the USA for use in patients aged 2 years and older, marking a significant milestone in its development journey.
$100 - $150
Hourly Rate
Trofinetide for Rett Syndrome: Highlights on the Development and Related Inventions of the First USFDA-Approved Treatment for Rare Pediatric Unmet Medical Need.
London, UK
2 hours ago
1 Received
- - Rett syndrome (RTT) is a rare neurodevelopmental disorder affecting girls, with no effective treatments approved by the USFDA until March 2023, when Trofinetide was finally authorized as the first medication for RTT.
- - Trofinetide, originally discovered in 2000 by Neuren Pharmaceuticals, is based on a neuroprotective peptide and aims to enhance neuronal structure and function, although its exact mechanism is still unclear.
- - The approval of Trofinetide marks a significant advancement in RTT treatment, opening doors for further research and clinical trials on related disorders like Fragile X syndrome and autism spectrum disorder.
$100 - $150
Hourly Rate
Trofinetide in Rett syndrome: A brief review of safety and efficacy.
London, UK
2 hours ago
1 Received
- * There is no cure for RTT, but symptoms can be managed with therapy; Trofinetide, initially developed for stroke, has shown positive results in Phase II clinical trials for RTT.
- * Recently, Trofinetide received FDA approval and orphan drug designation as the first approved treatment for RTT, demonstrating safety and effectiveness without known drug interactions.
$100 - $150
Hourly Rate
Trofinetide-a new chapter in rett syndrome's treatment.
London, UK
2 hours ago
1 Received
- - Trofinetide is the first FDA-approved medication for treating Rett Syndrome in children aged 2 years and older.
- - Clinical studies show that Trofinetide significantly improves behavioral scores related to Rett syndrome.
- - While more research is needed on potential side effects, Trofinetide represents a major advancement in treatment options for this condition.
$100 - $150
Hourly Rate
Development of trofinetide for the treatment of Rett syndrome: from bench to bedside.
London, UK
2 hours ago
1 Received
- * Trofinetide is a medication developed specifically for RTT, recently approved by the FDA for patients aged 2 years and older.
- * The successful development of trofinetide highlights the importance of collaboration between research institutions, pharmaceutical companies, and patient advocacy groups in creating effective treatments for rare diseases.
$100 - $150
Hourly Rate
Rett Syndrome and the Role of MECP2: Signaling to Clinical Trials.
London, UK
2 hours ago
1 Received
- Rett syndrome (RTT) is a neurological disorder primarily affecting females, with occurrences of 1 in 10,000 to 20,000 live births, leading to symptoms like hand movement stereotypes, learning impairments, and reduced lifespan.
- The most common causes of death in RTT patients are pneumonia and cardiorespiratory issues, with a 77.8% survival rate at 25 years and potential to live into their 50s.
- 95% of RTT cases are linked to mutations in the MECP2 gene, which regulates gene expression, and recent research is focusing on therapeutics targeting this gene.
$100 - $150
Hourly Rate
2023 FDA TIDES (Peptides and Oligonucleotides) Harvest.
London, UK
2 hours ago
1 Received
- In 2023, the FDA approved nine TIDES, including four oligonucleotides targeting disorders like ALS, geographic atrophy, primary hyperoxaluria type 1, and hereditary transthyretin-mediated amyloidosis, all with enhanced stability and effectiveness.
- The approved peptides exhibit a variety of structures (linear, cyclic, and lipopeptides) and applications, including the first orphan drug designation for a peptide-based chemokine antagonist.
- Notably, a peptide-based treatment was approved for core symptoms of Rett syndrome, with the analysis of TIDES focusing on their chemical structure, medical targets, and potential side effects.
$100 - $150
Hourly Rate
Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.
London, UK
2 hours ago
1 Received
- - Trofinetide, approved for treating Rett syndrome in March 2023, was tested in a phase 3 study to evaluate its impact on communication abilities in females aged 5 to 20, with results showing caregivers prioritizing communication improvements.
- - Participants were given either trofinetide or a placebo for 12 weeks, with communication outcomes measured using caregiver and clinician assessments, including the CSBS-DP-IT Social Composite score and ratings for nonverbal and verbal communication.
- - Results indicated trofinetide significantly improved caregiver-rated communication compared to placebo, particularly in the CSBS-DP-IT score, suggesting its potential value for future research on communication in Rett syndrome and related disorders.
$100 - $150
Hourly Rate
A Physiologically Based Pharmacokinetic Modeling Approach to Assess the Potential for Drug Interactions Between Trofinetide and CYP3A4-Metabolized Drugs.
London, UK
2 hours ago
1 Received
- Trofinetide is the first FDA-approved drug for treating Rett syndrome and is metabolized primarily by CYP3A4, which has low potential for interactions in the liver due to its high inhibitory concentration.
- The study utilized a physiologically based pharmacokinetic (PBPK) model to assess the effects of trofinetide on midazolam, a substrate for CYP3A4, and found that coadministration did not significantly alter midazolam's pharmacokinetic properties.
- However, it suggested that while trofinetide may weakly inhibit CYP3A4, it could enhance midazolam's absorption and bioavailability when taken together orally, indicating a potential for intestinal drug interactions.
$100 - $150
Hourly Rate
Safety and efficacy of trofinetide in Rett syndrome: a systematic review and meta-analysis of randomized controlled trials.
London, UK
2 hours ago
1 Received
- Rett syndrome is a rare genetic disorder that mainly affects females, characterized by the loss of skills, communication difficulties, and repetitive hand movements, highlighting the need for effective treatments.
- The study aimed to assess the safety and effectiveness of trofinetide for treating Rett syndrome through Randomized Controlled Trials, analyzing a total of 59 articles and including three eligible studies for in-depth review.
- Results indicated that trofinetide significantly improved behavior and clinical outcomes at a 200 mg dosage, evidenced by better scores on the Rett Syndrome Behavior Questionnaire and Clinical Global Impression-Improvement, although no notable effects were recorded on motor skills or caregiver concerns.
$100 - $150
Hourly Rate
An Update on Psychopharmacological Treatment of Autism Spectrum Disorder.
London, UK
2 hours ago
1 Received
- Behavioral interventions are the primary treatment for autism spectrum disorder (ASD), but new targeted treatments addressing its neurophysiology are emerging.
- These treatments, influenced by genetic factors and the diverse causes of ASD, may include medications and even gene therapies in the future.
- The article reviews current pharmacological options for ASD, including common medications for related issues and potential new treatments like metformin and cannabidiol.
$100 - $150
Hourly Rate
Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome.
London, UK
2 hours ago
1 Received
- - Trofinetide was recently approved for treating Rett syndrome based on positive results from the phase 3 LAVENDER study, which allowed for a weight-based dosing approach.
- - Efficacy endpoints like the Rett Syndrome Behaviour Questionnaire (RSBQ) and Communication and Symbolic Behavior Scales (CSBS-DP-IT) showed significant improvement correlating with higher trofinetide drug exposure.
- - The analysis confirmed that higher drug exposure within the target range significantly improved symptoms in patients, supporting the established dosing regimen for trofinetide.
$100 - $150
Hourly Rate
Managing Gastrointestinal Symptoms Resulting from Treatment with Trofinetide for Rett Syndrome: Caregiver and Nurse Perspectives.
London, UK
2 hours ago
1 Received
- Rett syndrome is a rare neurodevelopmental disorder that primarily affects females, and trofinetide is the first FDA-approved treatment based on positive results from the phase 3 LAVENDER trial.
- Despite its benefits in managing core RTT symptoms, trofinetide has side effects, notably gastrointestinal issues like diarrhea and vomiting.
- The article explores insights from caregivers of girls undergoing trofinetide trials and nurse coordinators, emphasizing strategies for managing these GI symptoms during treatment.
$100 - $150
Hourly Rate
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Look at DAYBUE™ (Trofinetide) as first ever drug for treatment of Rett Syndrome.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
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Trofinetide receives FDA approval as first drug for Rett syndrome.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Recommendations for the management of gastrointestinal comorbidities with or without trofinetide use in Rett syndrome.
London, UK
2 hours ago
1 Received
- - Over 90% of individuals with Rett syndrome (RTT) experience gastrointestinal (GI) comorbidities, but many healthcare providers do not consider managing these issues a top priority in treatment.
- - Trofinetide, the first drug approved for RTT, has shown improvements in symptoms but can lead to GI side effects like diarrhea and vomiting, highlighting the need for better management strategies.
- - The authors advocate for a proactive approach to treating GI issues in RTT patients, using both clinical experience and literature recommendations to improve drug tolerance and enhance quality of life.
$100 - $150
Hourly Rate
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Trofinetide (Daybue) for Rett syndrome.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Is trofinetide a future treatment for Rett syndrome? A comprehensive systematic review and meta-analysis of randomized controlled trials.
London, UK
2 hours ago
1 Received
- Rett syndrome (RTT) is a rare genetic disorder that poses significant treatment challenges, and trofinetide has shown promise in improving symptoms in affected individuals.
- A systematic review and meta-analysis of three randomized controlled trials involving 276 patients indicated that trofinetide significantly improved caregiver outcomes and overall clinical measures, particularly in the Rett Syndrome Behavior Questionnaire and Clinical Global Impression Scale.
- While trofinetide showed benefits, it was associated with increased risks of vomiting and diarrhea compared to placebo, highlighting the need to consider its safety profile in clinical use.
$100 - $150
Hourly Rate
Insulin-Like Growth Factor 1 Has the Potential to Be Used as a Diagnostic Tool and Treatment Target for Autism Spectrum Disorders.
London, UK
2 hours ago
1 Received
- Autism spectrum disorder (ASD) is a complex neurodevelopmental condition marked by social challenges and repetitive behaviors, making diagnosis and treatment difficult due to the absence of specific lab tests.
- Research indicates that levels of insulin-like growth factor 1 (IGF-1) are lower in preschoolers with ASD compared to their peers, suggesting it could serve as a diagnostic marker.
- IGF-1 treatment has shown promise in improving core symptoms and underlying brain issues in various ASD models, and a derivative of IGF-1 was recently approved to treat Rett syndrome, an ASD-related disorder, highlighting its potential both as a diagnostic tool and a therapy for ASD.
$100 - $150
Hourly Rate
Effect of Hepatic Impairment on Trofinetide Exposures Using an In Silico Physiologically Based Pharmacokinetic Model.
London, UK
2 hours ago
1 Received
- Trofinetide is the first approved treatment for Rett syndrome, and the study examines how liver impairment affects its pharmacokinetics (PK).
- Researchers used modeling to analyze drug concentration in virtual patients with varying degrees of liver impairment compared to healthy individuals after taking a 12 g dose.
- The results showed that liver impairment doesn't significantly impact trofinetide exposure, though slight increases in drug concentration were observed with more severe liver conditions due to hematocrit changes.
$100 - $150
Hourly Rate