Added 1 day ago Drug news

Phase III study of Brabafen in Dravet Syndrome published in JAMA Neurology.- Zogenix

Using Brabafen (fenfluramine) from Zogenix in an antiseizure medication regimen that includes the anticonvulsant stiripentol significantly reduces seizures in Dravet...

Added 22 days ago Drug news

GW Pharmaceuticals receives positive NICE recommendation for Epidyolex for the treatment of seizures in patients with two rare, severe forms of childhood-onset epilepsy.

GW Pharmaceuticals plc announces that two of its medicines, Epidyolex (cannabidiol) oral solution and Sativex (nabiximols), have been recommended by...

Added 24 days ago Drug news

Phase III P301 trial of SPN 810 in patients with ADHD, did not meet its primary endpoint.- Supernus Pharma

Supernus Pharmaceuticals, Inc. reported financial results for the third quarter of 2019, results from the Phase III P301 trial for SPN...

Cannabis-based medicinal products

This guideline covers prescribing of cannabis-based medicinal products for people with intractable nausea and vomiting, chronic pain, spasticity and severe treatment-resistant epilepsy.

Added 18 hours ago

Fever in under 5s: assessment and initial management

This guideline covers the assessment and early management of fever with no obvious cause in children aged under 5. It aims to improve clinical assessment and help healthcare professionals diagnose serious illness among young children...

Added 6 years ago

Epilepsies: diagnosis and management

The guideline covers diagnosing, treating and managing epilepsy and seizures in children, young people and adults in primary and secondary care.

Added 7 years ago

Advances in gene therapy for cystic fibrosis lung disease.

Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung...

Added 7 days ago

Children With Cystic Fibrosis Are Infected With Multiple Subpopulations of Mycobacterium abscessus With Different Antimicrobial Resistance Profiles.

Background: Children with cystic fibrosis (CF) can develop life-threatening infections of Mycobacterium abscessus. These present a significant clinical challenge, particularly when the strains involved are resistant to antibiotics.

Added 7 days ago

Prevention of chronic infection with Pseudomonas aeruginosa infection in cystic fibrosis.

Purpose of review: This review provides an update on definitions of chronicity of infection, approaches to airway sampling to detect infection, strategies for Pseudomonas aeruginosa eradication, impact of cystic fibrosis...

Added 7 days ago

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

The primary objective of the study is to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA).

Added 2 months ago

A Study of Ocrelizumab in Children and Adolescents With Relapsing-Remitting Multiple Sclerosis

This 2-year study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) effects of ocrelizumab in children and adolescents ages >/= 10 to < 18 years with relapsing-remitting multiple sclerosis (RRMS).

Added 1 month ago

A Dose-finding Study of Inhaled OligoG vs Placebo in Patients With Cystic Fibrosis (SMR3372)

Randomized, double blind, placebo controlled study. The study has two parts:
Dose-finding part, followed by longer term follow-up (6 months)

Added 1 month ago