Data Demonstrates the Best Intervention for Pediatric Sleep Health is Earlier Bedtime
September 12th 2022Nonpharmacological sleep interventions that focused on earlier sleep schedules can serve as a “simple, pragmatic, effective way to meaningfully increase sleep duration that could have important benefits for child health.”
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Velmanase Alfa Therapy BLA Granted Priority Review by FDA for Alpha-Mannosidosis
September 12th 2022The FDA accepted the Biologics License Application (BLA) and granted priority review for velmanase alfa, an investigational enzyme replacement therapy intended to treat the ultra rare disease.
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Promising Data from InPedILD Phase 3 Trial Revealed at ERS Congress 2022
September 8th 2022Novel insights into the risk-benefit profile of nintedanib in children and adolescents with fibrosing interstitial lung disease (ILD) were provided by late-breaking data presented at ERS 2022 regarding the InPedILD clinical trial.
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Pharmacy-Supported Digital Medicine Program Improves Asthma Control
September 8th 2022Late-breaking data presented at ERS Congress 2022 show that inhaler sensors and mobile health (mHealth) applications combined with pharmacist counseling improved treatment adherence for patients with uncontrolled asthma.
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Telehealth Services Rendered During Pandemic Improved Opioid Use Disorder Care Retention
September 6th 2022CDC investigators stated that the expansion of telehealth strategies is “urgently needed” to provide medications for opioid use disorder because it could increase retention in care and improve outcomes for OUD.
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Phase 2b Study Supports Icenticaftor for Patients with COPD, Chronic Bronchitis
September 6th 2022“From this study it makes sense that the (icenticaftor) 300mg dose has a benefit-risk profile to further study the clinical benefits,” Dr. Frits Franssen, lead investigator, said at ERS Congress 2022.
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Efanesoctocog Alfa Attains FDA Priority Review for Treatment of Hemophilia A
August 31st 2022The FDA granted priority review for the Bioloigics License Application (BLA) Sanofi submitted for efanesoctocog alfa indicated for individuals with hemophilia A with a decision date set for February 28, 2023.
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Rare Disease Report Podcast: CDKL5 Deficiency Disorder
August 31st 2022In the first episode of a 2-part Rare Disease Report, Whitney Mitchell, mother and caregiver to her 5 year old daughter Havilah, shared her experience leading up to, and now living with, Havilah’s CDKL5 deficiency disorder diagnosis.
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