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http://dx.doi.org/10.3324/haematol.2023.283992 | DOI Listing |
Am J Respir Cell Mol Biol
January 2025
Ottawa Hospital Research Institute & CHEO Research Institute, Pediatrics, Ottawa, Ontario, Canada.
Surfactant protein-B (SP-B) deficiency is a lethal neonatal respiratory disease with few therapeutic options. Gene therapy using adeno-associated viruses (AAV) to deliver human cDNA (AAV-hSPB) can improve survival in a mouse model of SP-B deficiency. However, the effect of this gene therapy wanes.
View Article and Find Full Text PDFJ Am Acad Orthop Surg Glob Res Rev
January 2025
Universidad Autónoma de Guadalajara, School of Medicine, Zapopan, Mexico.
Background: Physicians worldwide face the challenging task of improving patient satisfaction by reducing pain in injured patients. Currently, available therapeutic approaches provide only short-term relief of symptoms without addressing long-term satisfaction. This has led to exploring regenerative treatment options that can deliver better outcomes.
View Article and Find Full Text PDFJ Infect Dis
January 2025
Department of Oral and Craniofacial Biology, School of Dentistry, LSU Health New Orleans, USA.
Background: Vulvovaginal candidiasis (VVC), caused primarily by Candida albicans, is currently treated with either prescription or over-the-counter antifungal drugs, often with variable efficacy and relapses. New and improved therapeutic strategies, including drug-free treatment alternatives, are needed. Upon overgrowth or environmental triggers, C.
View Article and Find Full Text PDFPLoS One
January 2025
Department of Medicine, Division of Pulmonary Medicine, University of Alberta, Edmonton, AB, Canada.
Primary and secondary antibody deficiencies (PAD and SAD) are amongst the most prevalent immunodeficiency syndromes, often necessitating long-term immune globulin replacement therapy (IRT). Both intravenous immunoglobulin (IVIG) and subcutaneous immunoglobulin (SCIG) have demonstrated efficacy in antibody deficiency. Comparative analyses of these two routes of administration are limited to nurse-administered IVIG and home therapy with self-administered SCIG.
View Article and Find Full Text PDFJAMA Neurol
January 2025
Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
Importance: There is a lack of long-term efficacy and safety data on hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) and on RNA interference (RNAi) therapeutics in general. This study presents the longest-term data to date on patisiran for hATTR-PN.
Objective: To present the long-term efficacy and safety of patisiran in adults with hATTR-PN.
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