Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam led to rapid and sustained increases in SMN protein levels. No new safety signals were observed and the overall adverse event profile was consistent with that of treatment-naive patients.“These 24 month exploratory data are important as they are consistent with the medically meaningful results we saw after one year in Part 2 of the SUNFISH study, designed to represent a broad, real-world SMA population,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are also encouraged to see an increase in SMN protein levels across both the Sunfish Part 1 and Jewelfish studies. These data reinforce the potential of risdiplam to make a real difference in the lives of the many people living with SMA.” SUNFISH is a large (n=231) global two-part study in children and adults. The dose-findi...
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...
We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice. EXPERT OPINION: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries. PMID: 33034214 [PubMed - as supplied by publisher]
CONCLUSIONS: Xyloglucan/gelose plus ORS was effective and safe in treating acute diarrhea in children. PMID: 33028102 [PubMed - as supplied by publisher]
Currently in fellowship doing bread/butter procedures (MBB, epidurals, PNB, few SCS/PNS trials, etc.) and just interviewed at a private practice spot where they do a lot of procedures that I will have not done any training in prior to graduating (e.g. IT pump, SI fusion, Vertiflex, Kypho, MILD, Discectomy, lots of SCS/PNS trials etc) and significant amount of "OR pain procedures" at a very busy practice seeing 30-40 pts/day - how many of you are commonly performing these procedures and are... private practice concern
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
Publication date: October 2020Source: Brain, Behavior, and Immunity, Volume 89Author(s): Fernando Lopes, Fernando A. Vicentini, Nina L. Cluny, Alexander J. Mathews, Benjamin H. Lee, Wagdi A. Almishri, Lateece Griffin, William Gonçalves, Vanessa Pinho, Derek M. McKay, Simon A. Hirota, Mark G. Swain, Quentin J. Pittman, Keith A. Sharkey
BEST supplements to relieve joint pain: Is your arthritis playing up? The wetter and colder months could partially be to blame. These two pills may help.
Authors: Kim H, Lim YM, Lee EJ, Kim HW, Ahn HS, Kim KK PMID: 33029979 [PubMed]
CONCLUSIONS: More than half of the iIONP patients had an enhanced oculomotor nerve in MRI. A few of them also had elevated CSF IgG synthesis rate, but no further evidence for inflammation was found. The administration of steroids seemed to have no benefit other than increasing the blood glucose level. PMID: 33029972 [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]
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