Use of the basic form of recombinant human erythropoietin in the treatment of patients with spinocerebellar ataxia with CAG repeat mutations

• Main Authors: Velázquez Pérez, Luis Clodoaldo, Sosa Testé, Iliana María, López Matilla, Lien, García Rodríguez, Julio César, González Triana, Consuelo, Ortega Sánchez, Ricardo, Hernández Casaña, Patricia, Rodríguez Labrada, Roberto, Amaro González, Daniel Enrique, Jiménez Rodríguez, Daise, Jay Pérez, Daniel
• Format: Patent
• Language: English
• Published: 07.02.2019
• Subjects: HUMAN NECESSITIES; HYGIENE; INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIRCHEMICAL OR PHYSICAL PROPERTIES; MEASURING; MEDICAL OR VETERINARY SCIENCE; PHYSICS; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; TESTING

The present invention relates to the use of pharmaceutical compositions containing the basic form of recombinant human erythropoietin (rhEPO) as an active ingredient, which are administered via the nasal route, in the treatment of spinocerebellar ataxia (SCA) with CAG repeat mutations, particularly type 2 SCA. The invention also relates to a method for classifying patients as responsive or unresponsive to treatment with the basic form of rhEPO.