Several studies indicate that prognosis for survival in Duchenne muscular dystrophy (DMD)
has improved in recent decades. However, published evidence is inconclusive and some …

Background Duchenne muscular dystrophy (DMD) is a severe rare progressive inherited
neuromuscular disorder, leading to loss of ambulation (LOA) and premature mortality. The …

In France, the epidemic phase of COVID-19 caused by severe acute respiratory syndrome
coronavirus 2 (SARS-CoV-2) began in February 2020 and resulted in the implementation of …

Aim To conduct a systematic literature review of caregiver burden in Duchenne muscular
dystrophy (DMD). Method We searched Embase, Web of Science, and PubMed for full‐text …

We report a first-in-patient study of vamorolone, a first-in-class dissociative steroidal anti-
inflammatory drug, in Duchenne muscular dystrophy. This 2-week, open-label Phase IIa …

Abstract Objective Strategic Targeting of Registries and International Database of
Excellence (STRIDE) is an ongoing, international, multicenter registry of real-world ataluren …

Objective This systematic review aims to update the evidence on Duchenne muscular
dystrophy (DMD) in Italy, describing the epidemiology, quality of life (QoL) of patients and …

Methods The Delphi Consensus Initiative presented here is focused on how to reduce the
time to diagnosis of DMD. The development process for this initiative is summarized in …

Anesthetic consideration for neuromuscular diseases : Current Opinion in Anesthesiology
Anesthetic consideration for neuromuscular diseases : Current Opinion in Anesthesiology Log …

Background: Noninvasive mechanical ventilation (NIV) is an effective treatment in patients
with neuromuscular diseases (NMD) to improve symptoms, quality of life, and survival …