Selecting Cognitive & Behavioral Endpoint Measures for Trials in...
Selecting cognitive and behavioral endpoints for rare epilepsy and mucopolysaccharidoses (MPS) trials can be challenging. These diseases are often regressive, making the assessment of cognition and...(PRWeb November 05, 2019)Read the full story at https://www.prweb.com/releases/selecting_cognitive_behavioral_endpoint_measures_for_trials_in_rare_epilepsy_and_mps_disorders_upcoming_webinar_hosted_by_xtalks/prweb16698077.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 5, 2019 Category: Pharmaceuticals Source Type: news

Simple sugar delays neurodegeneration caused by enzyme deficiency
(Baylor College of Medicine) The sugar trehalose increases cellular waste disposal and improves the neurological symptoms in a mouse model of mucopolysaccharidoses IIIB . (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - June 20, 2018 Category: Biology Source Type: news

Medical first: Doctors attempt to gene-edit a living patient
A man may soon be forever free of the previously incurable disease he was born with 44 years ago. On Monday, in a medical first, Brian Madeux received an experimental in-body gene-editing treatment intended to cure him of Mucopolysaccharidoses II, known as MPS II or Hunter syndrome, a rare disorder that causes progressive damage to the body's cells. (Source: CNN.com - Health)
Source: CNN.com - Health - November 16, 2017 Category: Consumer Health News Source Type: news

Medical first: Doctors attempt to gene-edit a living patient's DNA
A man may soon be forever free of the previously incurable disease he was born with 44 years ago. On Monday, in a medical first, Brian Madeux received an experimental in-body gene-editing treatment intended to cure him of Mucopolysaccharidoses II, known as MPS II or Hunter syndrome, a rare disorder that causes progressive damage to the body's cells. (Source: CNN.com - Health)
Source: CNN.com - Health - November 16, 2017 Category: Consumer Health News Source Type: news

New biomarker found for group of rare metabolic diseases
(University of Pennsylvania School of Medicine) A newly discovered biomarker associated with a rare metabolic disorder may facilitate better diagnosis and identification of new drugs for clinical trials for the disease. Development of treatments for the neurological symptoms of mucopolysaccharidoses, a family of rare genetic disorders, have been hindered by the lack of objective measures of the extent of central nervous system damage in patients. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 7, 2017 Category: International Medicine & Public Health Source Type: news

What Causes Muscle Weakness?
Discussion Muscle tone is the slight tension that is felt in a muscle when it is voluntarily relaxed. It can be assessed by asking the patient to relax and then taking the muscles through a range of motion such as moving the wrists, forearm and upper arm. Muscle strength is the muscle’s force against active resistance. Impaired strength is called weakness or paresis. There are 5 levels of muscle strength. 0 = No muscle contraction detected 1 = Barely detected flicker of contraction 2 = Active movement with gravity eliminated 3 = Active movement against gravity 4 = Active movement against gravity and some resistance ...
Source: PediatricEducation.org - December 9, 2013 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Orthopaedic Surgery for MPS DisordersOrthopaedic Surgery for MPS Disorders
Learn more on the clinical findings in mucopolysaccharidoses and treatment strategies for patients with these disorders. Current Orthopaedic Practice (Source: Medscape Orthopaedics Headlines)
Source: Medscape Orthopaedics Headlines - February 8, 2013 Category: Orthopaedics Tags: Orthopaedics Journal Article Source Type: news