NICE recommends alternative corticosteroid for people with Duchenne muscular dystrophy The guidance says that vamorolone reduces “adverse events” that are tied to other currently available corticosteroids, which include osteoporosis, weight gain and delayed puberty.…
Paediatric arrhythmias: pathophysiology, types and pharmaceutical management While many arrhythmias in children are harmless, it is vital to distinguish between the different types to ensure that potentially harmful ones are identified early and treatment plans are optimised for the patient.…
Declining use of sodium valproate linked to fall in birth defects among children, study results show Children exposed to levetiracetam, oxcarbazepine and lamotrigine in the womb had the lowest prevalence of major congenital malformations.…
NHS to start routinely offering blindness drug to premature babies Treatment with ranibizumab, which is administered using a precision injection system, would begin two or three days after diagnosis of retinopathy of prematurity.…
Government-funded research to screen genomes of 100,000 babies for rare diseases The Newborn Genomes Programme, which will begin in 2023, will sequence the genomes of newborn babies to screen for rare conditions, to assess whether whole genome sequencing should be part of routine care.…
Clinical impact of CFTR modulator therapy roll-out An overview of the essential role a specialist cystic fibrosis pharmacist has in the optimisation of CFTR modulator combinations and a closer look at real-world clinical outcomes.…
Project to sequence 100,000 genomes from NHS patients hits target The 100,000 Genomes Project has reached its goal to sequence 100,000 genomes from NHS patients by the end of 2018, Matt Hancock, health and social care secretary, has announced.…
FDA approves new treatment for young children with Batten disease The first drug of its kind to be made available in the United States for children with a specific form of the inherited disorder, Batten disease, has been approved by the US Food and Drug Administration (FDA).…
Stem-cell screening finds statin alternative for hypercholesterolaemia Researchers discover a stem-cell based approach that may help identify new treatments for inherited liver diseases…
EMA recommends first EU treatment for spinal muscular atrophy The European Medicines Agency (EMA), which evaluates medicinal products for use in EU, has recommended nusinersen (Spinraza; Biogen) as a treatment for patients with spinal muscular atrophy (SMA). There is currently no approved therapy in the EU for SMA, a rare and often fatal genetic disease …
