July 2nd 2025
Additionally, long sleep duration could be associated with increased odds of being reliably symptomatic at 4 weeks.
June 18th 2025
The patient described the headache as “pressure-like,” episodes that occurred for 1 to 2 hours with high severity.
Currently, omaveloxolone (Skyclarys) is approved to treat adults and adolescents aged 16 years and older impacted by FA.
May 1st 2025
In this article, we recap our top stories, expert interviews, and Q+A discussions from the 2025 Pediatric Academic Societies meeting.
April 26th 2025
New data from national survey highlights disparities and need for focused prevention strategies.
Pegtarazimod data confirms inflammation biomarkers in hypoxic ischemic encephalopathy infants
In addition, data presented at PAS revealed a predictable pharmacokinetic profile in the newborn population, consistent with adult humans.
FDA expands diazepam nasal spray for seizure clusters to ages 2–5
Diazepam nasal spray (Valtoco) is approved for short-term treatment of seizure clusters, now in patients aged 2 years and older.
Preterm skin-to-skin contact does not improve neurodevelopment at 2–3 years
In the immediate skin-to-skin contact group, improved breastfeeding practices up to 12 months were observed.
Preterm birth associated with long-term cognitive issues in children
Infants born moderately preterm had lower composite cognitive scores compared to those born at full-term s (β = −0.39; 95% CI, −0.55 to −0.22; P < .001).
FDA accepts sBLA to expand fremanezumab-vfrm indication for episodic migraines in children
If approved, the expanded indication would include children and adolescents aged 6 to 17 years who weigh 99 lbs (45 kg) or more.
Navepegritide NDA submitted to treat children with achondroplasia
Submission is based on 3 clinical trials, including data from the ApproaCH Trial among children with achondroplasia.
FDA approves diazoxide choline extended-release tablets for hyperphagia in Prader-Willi syndrome
Diazoxide choline has been granted breakthrough, fast track, and orphan drug designations in the United States.
New questionnaire effectively screens for PANDAS/PANS
The PANDAS/PANS questionnaire is based on symptoms and comorbidities described in the literature and is a self-report completed by parents of children younger than 18.
OAV101 in SMA patients demonstrates improved motor function, disease progression
Results were from the phase 3 STEER study among a broad population of patients with SMA aged 2 to under 18 years.
FDA expands use of eculizumab to patients 6 years and up with generalized myasthenia gravis
First approved for PNH in 2007, the monoclonal antibody is now the first and only treatment for pediatric patients living with gMG.
FDA accepts new drug application for vatiquinone to treat Friedreich's ataxia
The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia, with a decision expected by August 19, 2025.
Weekly review: Several key FDA approvals, new puzzler case, and more
Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.
FDA approves non-invasive risdiplam tablet for spinal muscular atrophy
The new risdiplam tablet is suitable for those aged 2 years and older who weigh more than 44 lbs, according to Genentech.
Positive data reported for Trappsol Cyclo in patients 3 years and under with NPC1
Cyclo Therapeutics has enrolled 10 patients in a single-arm sub-study treating newborns to 3 years of age, evaluating the Trappsol Cyclo in the youngest subsets.
FDA news in pediatrics: January 2025
A recap of the FDA submissions and regulatory decisions in pediatrics from January 2025.
Apitegromab BLA submitted to FDA for spinal muscular atrophy
Application submission is based on positive phase 3 data that demonstrated improvement in patients with type 2 and 3 SMA receiving current standard of care.
FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy
The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months.
Study: No association between montelukast and neuropsychiatric adverse event risk
"In aggregation with other robust observational studies, these results can inform the management of asthma and allergic rhinitis in this patient group," wrote the study authors.
Using a smartphone application to screen for neonatal jaundice
A good correlation and statistical agreement with total serum bilirubin was observed in a study of a new smartphone-based machine learning app, leaving it a potential neonatal jaundice screening tool.
Survey: titrating trofinetide improves tolerability among Rett syndrome patients
Respondents reported decreased diarrhea severity, improved patient quality of life, and decreased treatment discontinuation among patients with Rett syndrome treated with trofinetide.
Fremanezumab for episodic migraine prevention demonstrates positive phase 3 data
Both monthly migraine days and headache days were reduced vs placebo in patients aged 6 to 17 years.
Saol Therapeutics submits NDA for SL1009 to treat Pyruvate Dehydrogenase Complex Deficiency
If approved, SL1009 has the potential to be the first approved medication for PDCD, and would be available as an oral solution.
Study: Concussions slow aperiodic activity among high school football players
Results demonstrated that slowed aperiodic activity was present in brain areas linked to concussion symptoms such as impaired concentration and memory.
Weekly report: Phase 3 data for JNJ2113, expert discussions, and more
Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from last week, all in one place.
Nerivio REN wearable now FDA-cleared to treat migraines in children 8 years and up
With this expanded FDA clearance, the wearable becomes the first FDA-cleared, non-drug therapy to treat acute migraines in children.
FDA approves Kebilidi as first gene therapy to treat aromatic L-amino acid decarboxylase deficiency
With the decision, Kebilidi becomes the first FDA-approved gene therapy for AADC deficiency, and is indicated for adult and pediatric patients.
NGN-401 to treat Rett syndrome demonstrates positive interim data
Data stems from the first 4 patients dosed in a low-dose cohort of an ongoing phase 1/2 open-label trial.
Marinus discontinuing oral ganaxolone trial development to treat seizures in children
The TrustTSC trial did not achieve its primary endpoint of percent reduction in 28-day frequency of TSC-associated seizures.
FDA approves sodium oxybate to treat cataplexy, EDS in pediatric narcolepsy patients
The approved indication is for patients aged 7 years and up with narcolepsy to treat cataplexy or excessive daytime sleepiness.
Discussing genetic diagnoses with parents can be tricky
Parents had a variety of preferences and motivations for acquiring prognostic genetic information about their child, but 4 major findings emerged from parental interviews. Click to read more.