Paediatrics

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study.  The study found that four out of five patients diagnosed with Sanfilippo have continued to gain cognitive skills in line with development in healthy children after being given the […]

The National Institute for Health and Care Excellence (NICE) has published its Final Guidance recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales.   Translarna is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy aged two years and older who can walk.  The final […]

UCB’s Fintepla (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS).  It has received authorisation as an add-on therapy to other anti-epileptic medicines for patients two years of age and older.  The approval was based on safety and efficacy data from a global, […]

The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.  RPDs are designed to encourage the development of new drugs with high unmet medical needs, including rare diseases in children.  The […]

Tellus Therapeutics, a company developing treatments for newborns in the neonatal intensive care unit (NICU), announced the closing of a Series A financing round of $35 million led by the Perceptive Xontogeny Venture Fund.   The capital will be used to continue preclinical work conducted through the seed investment with the Xontogeny development team and to […]

PacBio, developer of sequencing solutions, has announced its HiFi sequencing technology will be used in a pilot project for the Children’s Rare Disease Cohorts Initiative (CRDC) at Boston Children’s Hospital.   Boston Children’s Hospital researchers will use HiFi whole-genome sequencing (HiFi WGS) for the purpose of investigating genetic and epigenetic variants associated with rare paediatric diseases. […]

A Phase III trial has demonstrated that Jardiance (empagliflozin) is the first SGLT2 inhibitor to show a reduction in blood sugar levels in children and adolescents with type 2 diabetes.  The DINAMO Phase III clinical trial demonstrated a statistically significant reduction in HbA1c with empagliflozin compared with placebo for children and adolescents aged 10-17 years […]

The UK Medicines & Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for Novartis’ Cosentyx (secukinumab) in two types of juvenile idiopathic arthritis (JIA), enthesitis-related arthritis (ERA), and juvenile psoriatic arthritis (JPsA).  The drug is approved alone or in combination with methotrexate (MTX) in patients six years and older whose disease has responded inadequately […]