Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)

Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p
Source: Roche Media News - Category: Pharmaceuticals Source Type: news

Related Links:

ConclusionVariations of pH occurred in the majority (79.7%) of patients admitted with COVID-19. The patients experienced all the type of acid –base disorders, notably metabolic and respiratory alkalosis were the most common alterations in this group of patients.
Source: International Urology and Nephrology - Category: Urology & Nephrology Source Type: research
AbstractAccurate staging and re-staging of cancer in children is crucial for patient management. Currently, children with a newly diagnosed cancer must undergo a series of imaging tests, which are stressful, time-consuming, partially redundant, expensive, and can require repetitive anesthesia. New approaches for pediatric cancer staging can evaluate the primary tumor and metastases in a single session. However, traditional one-stop imaging tests, such as CT and positron emission tomography (PET)/CT, are associated with considerable radiation exposure. This is particularly concerning for children because they are more sensi...
Source: Pediatric Radiology - Category: Radiology Source Type: research
big dreamer here: if you were starting out as a first year med student, what is the path you’d take to secure a dream job specializing in lung cancer and related environmental research/advocacy at an academic medical center? what about while adding on an MBA and wanting to combine academia with entrepreneurship to find better lung cancer therapies?
Source: Student Doctor Network - Category: Universities & Medical Training Authors: Tags: Medical Students (MD) Source Type: forums
Your heart rate is a measurement of the number of times per minute that your heart contracts or beats. Monitoring your heart rate is useful for tracking fitness and managing the risk factors of heart and circulatory diseases like diabetes. So, how do you measure your heart rate and what is a healthy heart rate?
Source: Daily Express - Health - Category: Consumer Health News Source Type: news
Source: BMJ Comments - Category: General Medicine Source Type: forums
ConclusionWe observed a significant reduction in mortality during the period 2008 –2017 in diabetes either as underlying cause of death or when all mentions of diabetes in the death certificate were considered.
Source: Journal of Endocrinological Investigation - Category: Endocrinology Source Type: research
ConclusionsOur study confirms and extends our knowledge on the tyrosine kinase inhibitors effects on thyroid, showing that thyroid autoimmunity is frequently observed in chronic myeloid leukemia patients on long-term therapy and is associated with a better oncological response.
Source: Journal of Endocrinological Investigation - Category: Endocrinology Source Type: research
The objective of this study is to explore the relationship between serum 25 ‐hydroxyvitamin-D(25-(OH)2D3) level and sweat function in patients with type 2 diabetes mellitus (T2DM).MethodsA cross-sectional study of 1021 patients with T2DM who underwent 25-(OH)2D3 level detections and sweat function tests was carried out. These individuals were divided into deficient groups (n = 154 cases), insufficient groups (n = 593 cases) and sufficient groups (n = 274 cases). Spearman correlation analysis and multivariate stepwise linear regression analysis were implemented to determine the as...
Source: Journal of Endocrinological Investigation - Category: Endocrinology Source Type: research
AbstractPurposeAlthough the gut microbiota (GM) are associated with various diseases, their role in gestational diabetes mellitus (GDM) remains uncharacterized. Further study is urgently needed to expose the real relationship between GM and GDM.MethodsWe performed a prospective study in 33 pregnant Chinese individuals [15, GDM; 18, normal glucose tolerance (NGT)] to observe the fecal microbiota by 16S rRNA gene amplicon sequencing at 24 –28 weeks of gestational age after a standard 75 g oral glucose tolerance test. Linear regression analysis was employed to assess the relationships between the GM and GDM cl...
Source: Journal of Endocrinological Investigation - Category: Endocrinology Source Type: research
Boris Epshteyn, an adviser for former President Donald Trump's 2020 presidential campaign, acknowledged Friday that he was part of the effort to prop up so-called "alternate electors" to support...
Source: Reuters: Health - Category: Consumer Health News Source Type: news
More News: Academies | Allergy & Immunology | Alzheimer's | Autism | Babies | Biology | Biotechnology | Brain | Brazil Health | Bronchiolitis | Cancer | Cancer & Oncology | Child Development | Children | Chile Health | China Health | Clinical Trials | Common Cold | Constipation | Diabetes | Diabetes Type 1 | Diarrhoea | Endocrinology | European Medicines Agency (EMA) | Food and Drug Administration (FDA) | Genetics | Hospitals | Indonesia Health | Infectious Diseases | International Medicine & Public Health | Japan Health | Men | Motor Neurone Disease | Multiple Sclerosis | Muscular Dystrophy | Neurology | Neuroscience | Opthalmology | Partnerships | Pediatrics | Pharmaceuticals | Pneumonia | Rare Diseases | Reflex Sympathetic Dystrophy | Respiratory Medicine | Russia Health | Science | South Korea Health | Spinal Muscular Atrophy | Study | Switzerland Health | Taiwan Health | USA Health | WHO