Description protocol used to monitor patients treated with gene therapy
نویسندگان
چکیده
Background: The spinal muscular atrophy (SMA) is an autosomal recessive hereditary neuromuscular disease, categorized into 4 types according to the severity. Type 2 considered intermediate form. There are three medication options approved by ANVISA for SMA treatment: Spinraza, Ridisplam and Zolgensma.
منابع مشابه
USE OF MYELOPEROXIDASE INDEX AS A SUITABLE TOOL TO MONITOR RESPONSE TO THERAPY IN PATIENTS WITH MEGALOBLASTIC ANEMIA
Repeated bone marrow examination was found to be of value in assessing response to treatment in megaloblastic anemia. The objective of this study was monitoring the response of megaloblastics to treatment, concerning the location of neutrophilic myeloperoxidase and myeloperoxidase index (MPXI) and their variation in megaloblastic erythroid progeny. It is possible to follow up megaloblastic ...
متن کاملPatients treated with orthodontic-myofunctional therapeutic protocol.
AIM The aim of this study is to report three cases that needed myofunctional and orthodontic treatment and the good results achieved after the therapy. Orthodontic treatment alone, in presence of bad habits, is not enough to solve the orthodontic issues, so it needs to be combined with myofunctional treatment.
متن کاملOutcome of Paraquat Poisoned Patients Treated with a Commonly Used Therapeutic Flowchart: A Case Series
Background: Paraquat poisoning is a medical emergency challenge due to its inherent severe toxicity and unavailability of specific antidote for it. In this paper, a series of patients who were treated according to a commonly used treatment flowchart are presented. Methods: This prospective observational study was carried out on paraquat poisoned patients admitted to District Hospital, Chamaraj...
متن کاملuse of myeloperoxidase index as a suitable tool to monitor response to therapy in patients with megaloblastic anemia
repeated bone marrow examination was found to be of value in assessing response to treatment in megaloblastic anemia. the objective of this study was monitoring the response of megaloblastics to treatment, concerning the location of neutrophilic myeloperoxidase and myeloperoxidase index (mpxi) and their variation in megaloblastic erythroid progeny. it is possible to follow up megaloblastic case...
متن کاملExperiences with Protocol Description
This paper presents three conclusions about the description of protocols, based on extensive experience: (1) Informal methods are inadequate for widely used protocols. (2) Lightweight formal methods are easy and useful. (3) Informal natural language cannot be trusted, but natural-language paraphrases of formal language can be trusted for certain purposes. None of these conclusions will be new o...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Arquivos De Neuro-psiquiatria
سال: 2023
ISSN: ['1678-4227', '0004-282X']
DOI: https://doi.org/10.1055/s-0043-1774426