Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Cerliponase Alfa Impresses for CLN2 Disease

Publish date:: September 17, 2019

Key clinical point: Children with a form of Batten disease treated via intraventricular infusion of cerliponase alfa for 3 years showed significantly less decline in motor and language function.

Major finding: A positive treatment response was seen in 83% of patients after 3 years.

Study details: This is an ongoing, open-label study of 23 children with ceroid lipofuscinosis type 2 disease treated with cerliponase alfa.

Disclosures: The study was funded by BioMarin Pharmaceutical, which markets Brineura. Dr. Trivisano was a subinvestigator in the trial.

Citation:

Trivisano M et al. IEC 2019, Abstract P333.

This Week's Must Reads

Annual WCC visits significantly limit asthma worsening,

Does daily inhaler monitoring improve asthma outcomes in children?,

More severe AD correlates with worse sleep health and attention problems in children,

COVID-19–induced drop in first measles vaccinations sparks resurgence concerns,

Shortcomings identified in study of acne videos on TikTok,

Must Reads in Neurology

Gene-replacement therapy shows promise in X-linked myotubular myopathy,

Ataluren delays disease milestones in patients with nonsense mutation DMD,

Newer DMTs are more effective than injectable DMTs in pediatric MS,

Study supports halting antiseizure medications after neonatal seizures,

Evaluating the impact of new pediatric brain tumor classifications,