EspeRare Foundation

Recent papers from EspeRare Foundation

1. Prenatal therapies: a Points to Consider framework for responsible innovationBMC Medical Genomics · 2025
2. Perspectives on the role of “-Omics” in predicting response to immunotherapyEuropean Journal of Cancer · 2025
3. Inhibition of Sodium/Hydrogen Exchanger‐1 in the Right Ventricle and Lung Dysfunction Induced by Experimental Pulmonary Arterial Hypertension in RatsJournal of the American Heart Association · 2025
4. Inhibition of Na/H exchanger-1 in the right ventricle and lung dysfunction induced by experimental pulmonary arterial hypertension in rats2024
5. Protocol for the Phase 2 EDELIFE Trial Investigating the Efficacy and Safety of Intra-Amniotic ER004 Administration to Male Subjects with X-Linked Hypohidrotic Ectodermal DysplasiaGenes · 2023
6. Rimeporide as a first- in-class NHE-1 inhibitor: Results of a phase Ib trial in young patients with Duchenne Muscular DystrophyPharmacological Research · 2020
7. Safety and immunogenicity of Fc‐EDA, a recombinant ectodysplasin A1 replacement protein, in human subjectsBritish Journal of Clinical Pharmacology · 2020
8. Natural history of X-linked hypohidrotic ectodermal dysplasia: a 5-year follow-up studyOrphanet Journal of Rare Diseases · 2020
9. A C-terminal cysteine residue is required for peptide-based inhibition of the NGF/TrkA interaction at nM concentrations: implications for peptide-based analgesicsScientific Reports · 2019