Caregiver Burden Tracks With Anxiety in Parents of Children With SMA Type 1
A cross-sectional study of 100 Turkish parents found that heavier caregiving burden was significantly associated with higher state and trait ...
Tag: spinal muscular atrophy
Serum Myostatin Tracks Disease Severity in Spinal Muscular Atrophy, Small Study Finds
A study of 37 SMA patients in Milan finds serum myostatin correlates with motor function and disease severity, while Smad3 ...
Experts Agree Ventilated SMA Patients Deserve Access to Disease-Modifying Therapies
A UK modified Delphi consensus of 19 experts has produced 22 statements recommending that patients with spinal muscular atrophy on ...
Blood tau proteins linked to muscle and motor measures in adult SMA
A retrospective multicentre study linked blood p-tau181 and p-tau217 to motor, respiratory and creatine kinase measures in SMA, with exploratory ...
Three FDA-Approved Therapies Have Transformed Spinal Muscular Atrophy, Yet Major Unmet Needs Remain
A comprehensive review details how three FDA-approved therapies have dramatically improved spinal muscular atrophy outcomes while highlighting persistent comorbidities and ...
From First Breath to Lifelong Care: How Newborn Screening Is Being Rebuilt Around Every Child
The Society for Pediatric Research's 2026 Mary Ellen Avery Award recognizes Wendy Chung's work building population-scale newborn screening systems, from ...
Million-Dollar Cures: Why Insurance Policy Now Decides Which Children Get Gene Therapy
A new Pediatric Research commentary argues that insurance coverage and reimbursement policy, not the underlying science, now determine which children ...
Long-Read Sequencing Steps Into the Clinic for Spinal Muscular Atrophy Diagnosis
A new clinical study shows that third-generation long-read sequencing matches conventional tests perfectly while revealing hidden carrier risks and gene ...
REM Sleep Holds the First Clues That Children With Muscle Disease Need Breathing Support
A Canadian multicenter study of 115 children shows that new CHEST guideline criteria and REM-sleep-specific measures detect early sleep-disordered breathing ...
3D Printing Enhances Ultrasound Guidance for Scoliosis Punctures
In an age where technology continually transforms medical practices, the integration of 3D printing into healthcare has gained considerable attention. ...
Flash mob in the nucleus
Study clarifies why some proteins 'flock together' in the nucleus Almost all cells in our body contain a nucleus: a ...
