Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older
Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi ™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease . SMA causes progressive muscle weakness and atrophy, and significant unmet need remains, particularly in adults living with this condition. The CHMP recommendation was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. A final decision regarding approval is expected from the European Commission in the next two months and will be applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was approved by the U.S. Food and Drug Administration (FDA) in August 2020.“Our close partnership with the SMA community has enabled the development of the first ‘at-home’ treatment for SMA in infants, children and adults with varying levels of disease severity, the majority of whom remain untreated,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Office r and Head of Global Product Development....
Authors: Rombauts A, Abelenda-Alonso G, Cuervo G, Gudiol C, Carratalà J Abstract INTRODUCTION: Despite adequate antibiotic coverage, community-acquired pneumonia (CAP) remains a leading cause of hospitalization and mortality worldwide. It induces both a local pulmonary and a systemic inflammatory response, particularly significant in severe cases. The intensity of the dysregulated host response varies from patient to patient and has a negative impact on survival and other outcomes. AREAS COVERED: This comprehensive review summarizes the pathophysiological aspects of the inflammatory response in CAP, brie...
Publication date: December 2020Source: Linguistics and Education, Volume 60Author(s): Virginia Langum, Kirk P.H. Sullivan
CONCLUSIONS: Xyloglucan/gelose plus ORS was effective and safe in treating acute diarrhea in children. PMID: 33028102 [PubMed - as supplied by publisher]
Cupressus macrocarpa leaves for biomonitoring the environmental impact of an industrial complex: The case of Puchuncaví-Ventanas in Chile. Chemosphere. 2020 Dec;260:127521 Authors: Gorena T, Fadic X, Cereceda-Balic F Abstract The Punchuncaví Valley is one of the most polluted areas in central Chile affected by anthropogenic emissions from the Ventanas Industrial Complex (IC) where the most important industry is the copper smelter and refinery. In this context, this research aims were to assess the usefulness of the Cupressus macrocarpa as a biomonitor. The leaf samples were taken from fi...
Publication date: Available online 9 October 2020Source: Neurología (English Edition)Author(s): N. Morollón, R. Belvís, A. De Dios, N. Pagès, C. González-Oria, G. Latorre, S. Santos-Lasaosa
CONCLUSIONS: The SMs in PWIBDs and a craniotomy performed immediately before starting the process of determining brain death seem to be related to lengthening the TT-BD. PMID: 33029974 [PubMed]
CONCLUSIONS: Neuro-ophthalmologic findings are mostly normal in patients with visual snow syndrome. Retinal or neurological diseases must be excluded as possible causes of visual snow. PMID: 33029971 [PubMed]
Publication date: Available online 10 October 2020Source: The Brazilian Journal of Infectious DiseasesAuthor(s): Ícaro Boszczowski, Francisco Chiaravalloti Neto, Marta Blangiardo, Oswaldo Santos Baquero, Geraldine Madalosso, Denise Brandão de Assis, Thais Olitta, Anna S. Levin
Publication date: Available online 10 October 2020Source: Journal of Acupuncture and Meridian StudiesAuthor(s): Natália Freire Valente, Eliezer de Sousa Cardoso, Juliana Alencar da Silva Resende, Jeferson Antônio Santos
Authors: Tamai H, Shingaki N, Ida Y, Shimizu R, Maeshima S, Okamura J, Kawashima A, Nakao T, Hara T, Matsutani H, Nishikawa I, Higashi K Abstract BACKGROUND: Although clinical use of sofosbuvir plus ribavirin has been approved for patients infected with genotype 2 hepatitis C virus, patients ≥ 75-years-old have not been included in previous clinical trials. AIM: To evaluate the real-world safety and efficacy of sofosbuvir plus ribavirin for elderly patients (≥ 75-years-old) compared to nonelderly patients, we conducted a post-marketing prospective cohort study. METHODS: We treated 265 patients with ge...
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