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Neffy Pre-Orders Launch, Trofinetide Data Highlighted, and Screen Time Impact on Sleep Assessed

• Pre-ordering has commenced for Neffy, an epinephrine nasal spray, offering a needle-free option for treating anaphylaxis in adults and children. • Trofinetide (Daybue) shows promise in treating Rett syndrome, with studies confirming its efficacy and safety in improving behavioral symptoms. • A study indicates that not all screen time before bed negatively impacts sleep in children, suggesting a need for revised recommendations. • Research suggests that fish intake during pregnancy is associated with a reduced likelihood of autism diagnosis in children.

ARS Pharmaceuticals has launched pre-ordering for Neffy, an epinephrine nasal spray designed to treat allergic reactions, including anaphylaxis, in both adults and pediatric patients weighing at least 66 pounds. This needle-free option aims to address the long-awaited demand for easier-to-administer epinephrine. Patients can pre-order Neffy through neffy.com and obtain the medication via a healthcare provider or virtual consultation, with delivery expected within two days of availability in the U.S.

Neffy: A Needle-Free Option for Anaphylaxis

Richard Lowenthal, Co-Founder and CEO of ARS Pharmaceuticals, highlighted that Neffy offers a significant advantage by eliminating the need for needles. The company is also providing assistance with insurance prior authorizations through neffy.com, potentially reducing co-pays to $25 for two devices. The neffyConnect service and BlinkRx online pharmacy are currently accepting prescriptions, with BlinkRx offering free shipping. Sung Poblete, CEO of Food Allergy Research and Education, emphasized the importance of timely epinephrine administration and reducing injection hesitancy for effective anaphylaxis management.

Trofinetide's Impact on Rett Syndrome

Alan Percy, MD, recently discussed advancements in treating Rett syndrome, emphasizing the FDA approval of trofinetide (Daybue; Acadia Pharmaceuticals) on March 13, 2023. This approval marked the first drug directly targeting Rett syndrome symptoms, a significant shift from previous medications that only addressed symptomatic care.

Clinical Trial Data

Dr. Percy highlighted the LAVENDER trial, a 12-week, double-blind, placebo-controlled study, which demonstrated significant improvements in the Rett Syndrome Behavioral Questionnaire (RSBQ) and Clinical Global Impression of Improvement (CGI-I). Two long-term open-label extension studies, LILAC-1 and LILAC-2, further confirmed the safety and efficacy of trofinetide in patients aged 5 to 22 years. While trofinetide has shown promise, common side effects include diarrhea and vomiting, sometimes leading to treatment discontinuation. However, gradual dose adjustments have improved tolerability over time.

Screen Time and Sleep Quality in Adolescents

A study published in JAMA Pediatrics has challenged the notion that all screen time before bed negatively impacts sleep in children aged 11 to 15 years. The study used an objective, night-by-night approach, tracking screen time and sleep quality in 79 participants using video cameras and wrist-worn accelerometers.

Key Findings

The results indicated that screen use in the two hours before bed did not significantly affect sleep duration or quality. However, using screens in bed, particularly for interactive activities like gaming, was associated with delayed sleep onset and reduced total sleep time. Each additional 10 minutes of interactive screen time in bed resulted in a 10-minute delay in sleep onset and a decrease in total sleep time. These findings suggest that presleep recommendations should be adjusted, as not all screen time before bed impairs sleep. The study aligns with concerns from the American Academy of Pediatrics about the impact of intense screen activities on sleep.

Prenatal Fish Intake and Autism Risk

Research published in The American Journal of Clinical Nutrition suggests that fish intake during pregnancy is linked to a reduced likelihood of autism diagnosis and autism-related traits in children. This association was not observed with omega-3 supplementation. The study, part of the Child Health Outcomes (ECHO) Program, analyzed data from 69 cohorts across the U.S., including 3,939 participants for fish intake analysis and 4,537 for omega-3 supplement use. Results showed that pregnant women who consumed fish had lower odds of having a child diagnosed with autism, while omega-3 supplement use had no significant impact. These findings support existing dietary guidelines that encourage fish consumption during pregnancy for its benefits, including reduced risks of autism-related outcomes. Researchers emphasized that fish with low toxicant levels should be prioritized.
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[1]
Weekly review: Neffy pre-ordering launches, trofinetide data, and more
contemporarypediatrics.com · Sep 7, 2024

Pre-ordering for epinephrine nasal spray (neffy) has launched for treating allergic reactions, including anaphylaxis. Th...

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