Portal de recerca
“Manejo terapéutico y situación actual de uso de recursos y costes de la Atrofia Muscular Espinal (AME) en España: análisis retrospectivo, multicéntrico de revisión de historias clínicas” Estudio TREaSURE.
Dades bàsiques
- Protocol:
- COAV101A1ES05
- EURDRACT:
- NCT:
- Centre:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Any inici:
- 2023
- Any de finalització:
- 2024
Documents
- No hi ha documents
Participants
Grups d'Investigació
Finançadors - Promotors
NOVARTIS FARMACEUTICA, S.A.
Resultats de l'Assaig Clínic
Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol
Rebollo, Pablo; (...); Vazquez-Costa, Juan F.
Article. 10.1007/s40120-023-00571-9. 2024
Do we really need to calculate a minimal important difference for ALSFRS-R?: A letter in response to 'Clinically meaningful change: evaluation of the Rasch-built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) and the ALSFRS-R' published in Vol. 24(3-4), pp. 311-316
Article. 10.1080/21678421.2023.2248199. 2024
Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population.
Borrego-Hernandez, Daniel; (...); Garcia-Redondo, Alberto
Article. 10.3390/biomedicines12020356. 2024
Lifesaving Treatments for Spinal Muscular Atrophy: Global Access and Availability
Armengol, Victor D.; (...); Roy, Bhaskar
Meeting Abstract. 2023
Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Armengol, Victor D; (...); Roy, Bhaskar
Article. 10.1212/CPJ.0000000000200224. 2024
Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen
Andres-Benito, Pol; (...); Povedano, Monica
Article. 10.3390/ijms25073810. 2024
Pain in Children and Adolescents with Spinal Muscular Atrophy: A Longitudinal Study from a Patient Registry.
Pitarch-Castellano I; (...); Madruga-Garrido M
Article. 10.3390/children10121880. 2023
Pilot study for the implementation of newborn screening for spinal muscular atrophy in Valencia
Berzal-Serrano, Alba; (...); Millan, Jose M.
Meeting Abstract. 10.2174/1871530323666230914122955. 2024
Risdiplam in non-sitter patients aged 16years and older with 5q spinal muscular atrophy.
Nungo Garzon, Nancy Carolina; (...); Vazquez-Costa, Juan F
Article. 10.1002/mus.27804. 2023