Background: The advent of disease-modifying treatments (DMT) has changed natural history in 5q Spinal muscular atrophy (SMA). The aim of this study was to report survival and functional aspects in all the Italian type I children born since 2016.
Methods: The study included all symptomatic children with type I SMA born since January 1st, 2016, when DMTs became available in Italy.
Purpose: The availability of care recommendations has improved survival and delayed the progression of clinical signs in Duchenne muscular dystrophy. The aim of the study was to perform a nationwide survey investigating the prevalence, age distribution, and functional status of Duchenne muscular dystrophyin Italy.
Methods: The survey was performed by collecting data from all 31 reference centers for Duchenne muscular dystrophy in Italy using a structured form.
Biologic medications have revolutionized the treatment of many dermatologic conditions. However, their use during pregnancy and breastfeeding is a subject of ongoing concern due to limited data on their safety in these populations. As the course of many inflammatory skin conditions is unpredictable during pregnancy and may worsen, biologics are important therapeutic tools for disease stabilization in this patient population.
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