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Exploring Novel Treatments for Rett Syndrome
STATEMENT OF NEED
Rett syndrome is a rare, debilitating neurodevelopmental disorder almost always associated with a spontaneous mutation in the methyl-CpG-binding protein 2 (MECP2) gene on the X-chromosome. Affected individuals experience loss of purposeful hand skills, abnormalities in gait, loss of spoken language, and stereotypic hand movements, with more severe manifestations including seizures, autistic features, autonomic nervous system dysfunction, breathing abnormalities, sleep dist... |
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Recurrent and Metastatic HNSCC: New Insights and Real-World Evidence for Improved Patient Outcomes
STATEMENT OF NEED
Head and neck cancers represent a heterogeneous group of tumors that originate in the lip/oral cavity, hypopharynx, oropharynx, nasopharynx, and larynx. An estimated 66,470 new cases are diagnosed in the United States annually, and 15,050 people die of the disease (Siegel et al, 2022). Approximately 90% of cases are classified as head and neck squamous cell carcinoma (HNSCC). Treatment of HNSCC is complicated by numerous factors, including differing therapeutic strategies ... |
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Management of Cancer Pain: Current Guidelines
STATEMENT OF NEED
Patients with cancer often suffer from a range of symptoms that negatively impact their quality of life. Among the most significant of these symptoms is pain, which can be caused by surgery, radiation, chemotherapy, targeted therapy, diagnostic procedures, and/or supportive care measures. Between 20% and 50% of patients with cancer experience pain, including moderate-to-severe pain in approximately 80% of patients with advanced-stage cancer (NCI, 2023). Despite awareness a... |
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