Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić +7 more
wiley +1 more source
The Toxic Effects of Hydrated Cement, Autoclaved Aerated Concrete, and Demolition Dusts on the Respiratory System in Rats. [PDF]
Kilic M +11 more
europepmc +1 more source
Abstract Aims This work assessed the pharmacokinetics (PK), safety and tolerability of glasmacinal (EP395, an oral anti‐inflammatory macrolide with negligible antimicrobial activity in development for COPD treatment) in two healthy participant trials: ‘first‐in‐human’ (FIH) and ‘drug–drug‐interaction’ (DDI).
Dave Singh +5 more
wiley +1 more source
Inhalation & intravenous: umbilical cord mesenchymal stem cell-derived exosomes therapy strategy for acute respiratory distress syndrome in a murine model. [PDF]
Yang L +8 more
europepmc +1 more source
Clarifying the neural circuit mechanisms of spontaneous social behavior in macaques. [PDF]
Ninomiya T +4 more
europepmc +1 more source
In vitro characterisation of fluticasone propionate and ciclesonide delivered with and without valved holding chambers using cascade impaction and an adult simulated breathing profile. [PDF]
Csonka LL, Lehtimäki L, Csonka P.
europepmc +1 more source
Molecular Hydrogen Attenuates Chronic Inflammation and Delays the Onset of Ultraviolet B-Induced Skin Carcinogenesis in Mice. [PDF]
Hori F +4 more
europepmc +1 more source
Mitochondrial Ultrastructure, Fission Proteins, Activity, and Motor Dysfunctions in the Innovative Parkinson's Disease Model Induced by Manganese Inhalation. [PDF]
Garcia-Caballero CA +13 more
europepmc +1 more source
Exploring clinician and patient perspectives on choice of general anaesthetic. A report of two U.S. surveys: an anesthesia clinician Multicenter Perioperative Outcomes Group survey and a patient Amazon Mechanical Turk survey. [PDF]
Holzer KJ +9 more
europepmc +1 more source

