The evidence for Cannabidiol is building
Cannabidiol has been licensed as an add-on treatment for seizures caused by tuberous sclerosis complex for patients aged 2 years and over. Clinical trial evidence shows that it reduces seizure frequency and increases seizure-free days compared to a placebo. While the cost-effectiveness estimates are uncertain, the benefits of reducing the severity of seizures and lowering the risk of sudden unexpected death in epilepsy make it an appropriate use of NHS resources. Therefore, it is recommended as a treatment option by NICE.
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PIONEER TEENS Trial Reveals Oral Semaglutide Diabetes Breakthrough for Pediatric Patients
Important results from the PIONEER TEENS phase 3a trial! Oral semaglutide diabetes therapy delivered statistically superior glycemic control compared with placebo in children and adolescents aged 10–17 years with type 2 diabetes. The trial met its primary endpoint with a 0.83% greater reduction i...
Terminal Complement Inhibition: Promising Results in IgAN from the I CAN Trial
Terminal complement inhibition with Ultomiris (ravulizumab) delivered a statistically significant and clinically meaningful reduction in proteinuria at week 34 in adults with immunoglobulin A nephropathy (IgAN) at high risk of progression. According to the prespecified interim analysis of the Pha...
Advancements in Rare Disease Therapies: CHMP’s April 2026 Insights and Economic Implications
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommendations from its 20–23 April 2026 meeting, included a particular focus on rare disease therapies. The CHMP issued positive opinions for five new medicines, three of which carry orphan designation, alo...