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The Standards Coordinating Body for Gene, Cell, and Regenerative Medicines and Cell-Based Drug Discovery (SCB) supports the development and commercialization of regenerative medicine products by identifying and addressing industry-wide challenges through standards. Through extensive stakeholder engagement, the implementation of rapid microbial testing methods (RMTMs) was identified as a high-priority need that must be addressed to facilitate more timely release of products. Since 2017, SCB has coordinated efforts to develop standards for this area through surveys, weekly meetings, workshops, leadership in working groups and participation in standards development organizations. This article describes the results of these efforts and discusses the current landscape of RMTMs for regenerative medicine products. Based on discussions with stakeholders across the field, an overview of traditional culture-based methods and limitations, alternative microbial testing technologies and current challenges, fit-for-purpose working to increase awareness of, dialog about and participation in efforts to develop standards in the regenerative medicine field.
Long-term outcomes of patients with mucopolysaccharidosis (MPS) VI treated with galsulfase enzyme replacement therapy (ERT) since infancy were evaluated.
The study was a multicenter, prospective evaluation using data from infants with MPS VI generated during a phase 4 study (ASB-008; Clinicaltrials.govNCT00299000) and clinical data collected ≥5 years after completion of the study.
Parents of three subjects from ASB-008 (subjects 1, 2, and 4) provided written informed consent to participate in the follow-up study. One subject was excluded as consent was not provided. Subjects 1, 2, and 4 were aged 0.7, 0.3, and 1.1 years, respectively, at initiation of galsulfase and 10.5, 7.9, and 10.5 years, respectively, at follow-up. All subjects had classical MPS VI based on pre-treatment urinary glycosaminoglycans and the early onset of clinical manifestations. At follow-up, subject 4 had normal stature for age; subjects 1 and 2 had short stature, but height remained around the 90th percentile of growth curves for and 4, and remained normal in subject 2.
Very early and continuous ERT appears to slow down the clinical course of MPS VI, as shown by preservation of endurance, functional dexterity, and several fine and gross motor competencies after 7.7-9.8 years of treatment, and less growth impairment or progression of cardiac disease than could be expected based on the patients' classical phenotype. ERT does not seem to prevent progression of skeletal or eye disease in the long term.
Very early and continuous ERT appears to slow down the clinical course of MPS VI, as shown by preservation of endurance, functional dexterity, and several fine and gross motor competencies after 7.7-9.8 years of treatment, and less growth impairment or progression of cardiac disease than could be expected based on the patients' classical phenotype. ERT does not seem to prevent progression of skeletal or eye disease in the long term.Cerebral palsy is the most common physical disability of childhood describing a heterogeneous group of neurodevelopmental disorders that cause activity limitation, but often are accompanied by disturbances of sensation, perception, cognition, communication and behavior, or by epilepsy. Inborn errors of metabolism have been reported in the literature as presenting with features of cerebral palsy. We reviewed and updated the list of metabolic disorders known to be associated with symptoms suggestive of cerebral palsy and found more than 150 relevant IEMs. This represents the fifth of a series of articles attempting to create and maintain a comprehensive list of clinical and metabolic differential diagnosis according to system involvement.
Current dosimetry protocols for clinical protons using air-filled ionization chambers assume that the perturbation correction factor is equal to unity for all ionization chambers and proton energies. selleck products Since previous Monte Carlo based studies suggest that perturbation correction factors might be significantly different from unity this study aims to determine perturbation correction factors for six plane-parallel and four cylindrical ionization chambers in proton beams at clinical energies.
The dose deposited in the air cavity of the ionization chambers was calculated with the help of the Monte Carlo code TOPAS/Geant4 while specific constructive details of the chambers were removed step by step. By comparing these dose values the individual perturbation correction factors p
, p
, p
, p
, p
⋅p
as well as the total perturbation correction factor p
were derived for typical clinical proton energies between 80 and 250MeV.
The total perturbation correction factor p
was smaller than unity for almost evionization chambers in proton beams were calculated using Monte Carlo simulations. In contrast to the assumption of current dosimetry protocols the total perturbation correction factor pQ can be significantly different from unity. Hence, beam quality correction factors [Formula see text] that are calculated with the help of perturbation correction factors that are assumed to be unity come with a corresponding additional uncertainty.Artificial intelligence (AI) refers to the ability of machines to perform intelligent tasks, and machine learning (ML) is a subset of AI describing the ability of machines to learn independently and make accurate predictions. The application of AI combined with "big data" from the electronic health records, is poised to impact how we take care of patients. In recent years, an expanding body of literature has been published using ML in cardiovascular health care, including mechanical circulatory support (MCS). This primer article provides an overview for clinicians on relevant concepts of ML and AI, reviews predictive modeling concepts in ML and provides contextual reference to how AI is being adapted in the field of MCS. Lastly, it explains how these methods could be incorporated in the practices of medicine to improve patient outcomes.
Website: https://www.selleckchem.com/products/4egi-1.html
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