Weekly summary: Gene therapy for profound genetic deafness, rise in measles cases, and more.

Published Date: 16 May 2024

Catch up on what has been going on in our journal this past week by going over some of the most popular stories that have been posted on the Contemporary Pediatrics website.

Read Full News

Latest News

One Shot or Two? What to Know About HPV Vaccination Recommendations

— Guidance is evolving

What's Behind Boy's Bullous Skin Lesions After Flu Symptoms?

Mycoplasma pneumonia identified as the culprit in this case of RIME

Meta's $17.1 Billion Settlement: A Chance for a Social Media Natural Experiment

Whether it advances the science depends on who can study the results

CDC, Epidemiologists Work on Definition for Measles Deaths

Standard workgroup process began over the summer

Navigating the Evolving Treatment Landscape in SMA: An Interactive Clinical Decision-Making Simulation

Navigating the Evolving Treatment Landscape in SMA: An Interactive Clinical Decision-Making Simulation

Navigating the Spectrum of SMA: From Genetic Insights to Timely Diagnosis

Navigating the Spectrum of SMA: From Genetic Insights to Timely Diagnosis

Todays Updates

Trappsol Cyclo Misses Primary End Point in Phase 3 Trial for Niemann-Pick Disease Type C Isabella Ciccone, MPH

Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.

NeurologyLive® Friday 5 — October 2, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 2, 2026.

Todays Updates

Trappsol Cyclo Misses Primary End Point in Phase 3 Trial for Niemann-Pick Disease Type C Isabella Ciccone, MPH

Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.

NeurologyLive® Friday 5 — October 2, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 2, 2026.

Latest News

— Guidance is evolving

Mycoplasma pneumonia identified as the culprit in this case of RIME

Whether it advances the science depends on who can study the results

Standard workgroup process began over the summer

Navigating the Evolving Treatment Landscape in SMA: An Interactive Clinical Decision-Making Simulation

Navigating the Spectrum of SMA: From Genetic Insights to Timely Diagnosis

News All Time

Vax Beliefs of HHS Deputy Secretary Nominee Probed at Hearing

Chris Klomp praised the MMR vaccine, but Sen. Wyden said Klomp's answers "created confusion"

Inquiry Slams Hospital for Failure to Protect Babies After Nurse's Murder Conviction

Concerns were kept quiet as babies fell ill at hospital where Lucy Letby worked

Twins Conjoined at Head Separated; Brain-Eating Amoeba Source; Mercury-Laden Lotion

Health news and commentary gathered by MedPage Today staff

Where Does Medicine's Responsibility for Human Trafficking End?

Public health is political, but it doesn't have to be partisan, says Nicholas Peoples, MD

RFK Jr. Picks New USPSTF Members

Under Kennedy's scrutiny, the preventive services panel hasn't met in more than a year

Mysterious Stomach Rash Had Unusual Source

It stemmed from prolonged contact with a certain device

RFK Jr. Bolsters Vaccine Skepticism at Children's Health Defense Conference

"We have started to ask the questions that you ... have been asking for years," he said

Doc Running for Congress Calls RFK Jr. 'Dangerous and Incompetent'

Community health and vaccination rates are big issues for California pediatrician

Stuttering: New Consensus Statement Highlights Neurobiology

New consensus guideline frames stuttering as a neurobiological condition, emphasizing that physicians play a crucial role in diagnosis and ongoing management.

NHS Alliance Warns ADHD, Autism Costs Spiralling

NHS leaders say soaring ADHD and autism assessment costs are forcing service cuts and disrupting long-term planning.

A Camp for Children With Brain Injuries Zips Ahead

A Pennsylvania camp gives children and young adults with brain injuries a place to connect, but federal funding for traumatic brain injury programs remains uncertain.

Meta Settlement Falls Short on Teen Mental Health Protections, Whistleblower Says

(This August 27 story has been republished to add an image, with no changes to text.)

Gabapentin Not Linked to Better Outcomes for Kids’ Migraine

For children hospitalized with migraine, gabapentin use wasn’t associated with less pain or fewer readmissions than no use in an observational study.

This 'Natural' Remedy Was Likely Culprit in Baby's Lead Poisoning

Clinicians should ask parents about all meds, traditional and complementary, expert advises

Are Suicide Attempts Contagious in Young People?

— "We know that suicidal behavior does not occur in a social vacuum," researcher says

Dravet Syndrome: Optimizing Early Diagnosis and Treatment Strategies

Dravet Syndrome: Optimizing Early Diagnosis and Treatment Strategies

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