BioBlast Pharma (Israel)

Recent papers from BioBlast Pharma (Israel)

1. Assessment of PABPN1 nuclear inclusions on a large cohort of patients and in a human xenograft model of oculopharyngeal muscular dystrophyActa Neuropathologica · 2022
2. Trehalose alleviates the phenotype of Machado–Joseph disease mouse modelsJournal of Translational Medicine · 2020
3. TAT ‐ MTS ‐ MCM fusion proteins reduce MMA levels and improve mitochondrial activity and liver function in MCM ‐deficient cellsJournal of Cellular and Molecular Medicine · 2017
4. Frataxin‐deficient neurons and mice models of Friedreich ataxia are improved by TAT ‐ MTS cs‐ FXN treatmentJournal of Cellular and Molecular Medicine · 2017
5. Analysis of Azithromycin Monohydrate as a Single or a Combinatorial Therapy in a Mouse Model of Severe Spinal Muscular AtrophyJournal of Neuromuscular Diseases · 2017
6. Intravenous Trehalose Improves Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Preliminary Results of 24 Weeks Open Label Phase 2 Trial (S28.004)Neurology · 2016
7. First Human Use of High Dose IV Trehalose: Safety, Tolerability and Pharmacokinetic Results from the Oculopharyngeal Muscular Dystrophy (OPMD) Therapy Trial (P7.068)Neurology · 2015