Position Statement:Emerging genetic therapies for rare disorders
نویسندگان
چکیده
منابع مشابه
Novel Cell-Based Therapies in Hepatic Disorders
In the recent decade, liver diseases with high mortality rate have became one of the major health problems worldwide. Liver transplantation is the most effective and the standard treatment for decompensated liver disease, but, shortage of available organs and inaccuracy in modeling of the liver diseases are the most limiting issues in treatment. Regenerative medicine and cell based therapy str...
متن کاملClinical exome sequencing for genetic identification of rare Mendelian disorders.
IMPORTANCE Clinical exome sequencing (CES) is rapidly becoming a common molecular diagnostic test for individuals with rare genetic disorders. OBJECTIVE To report on initial clinical indications for CES referrals and molecular diagnostic rates for different indications and for different test types. DESIGN, SETTING, AND PARTICIPANTS Clinical exome sequencing was performed on 814 consecutive ...
متن کاملNutritional therapies for mental disorders
According to the Diagnostic and Statistical Manual of Mental Disorders, 4 out of the 10 leading causes of disability in the US and other developed countries are mental disorders. Major depression, bipolar disorder, schizophrenia, and obsessive compulsive disorder (OCD) are among the most common mental disorders that currently plague numerous countries and have varying incidence rates from 26 pe...
متن کاملEmerging therapies for mitochondrial disorders
Mitochondrial disorders are a diverse group of debilitating conditions resulting from nuclear and mitochondrial DNA mutations that affect multiple organs, often including the central and peripheral nervous system. Despite major advances in our understanding of the molecular mechanisms, effective treatments have not been forthcoming. For over five decades patients have been treated with differen...
متن کاملEmerging therapies for cerebrovascular disorders.
New information about therapeutic interventions regarding several important aspects of cerebrovascular disease has appeared recently. This short review will focus on new therapeutic developments concerning the prevention of ischemic stroke, acute ischemic stroke therapy trials, and, lastly, the management of patients with intracranial aneurysms. Great strides have been made in the pharmacologic...
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: Journal of the International Child Neurology Association
سال: 2019
ISSN: 2410-6410
DOI: 10.17724/jicna.2019.172