Parent Projects

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MDSA Parent Projects


Parent Project Muscular Dystrophy is a not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker Muscular Dystrophy.
Even though Parent Project Muscular Dystrophy was founded by parents of children with Duchenne and Becker MD, and it’s mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by these dystrophies through research, advocacy, education and compassion, Maxine Strydom has extended this in South Africa to include all children who are affected with MD. contact Maxine Strydom, Tel: 083 662 2189, Email: maxinestrydom@dpgprop.co.za
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Neuropsychiatric Disorders in Males With Duchenne Muscular Dystrophy.


Frequency Rate of Attention-Deficit Hyperactivity Disorder (ADHD), Autism Spectrum Disorder, and Obsessive-Compulsive Disorder, Using a questionnaire-based study, we assessed the parentreported prevalence of attention-deficit hyperactivity disorders (ADHDs), autism spectrum disorders, and obsessive-compulsive disorders in a group of 351 males with Duchenne muscular dystrophy. Of the 351 males with Duchenne muscular dystrophy, 11.7% were reported to have a comorbid diagnosis of ADHD, 3.1% had autism spectrum disorder, and 4.8% had obsessive-compulsive disorder. It can be concluded that the incidence of these neuropsychiatric disorders is higher in Duchenne males than in the normal population. This finding, together with recent reports on the higher prevalence of cognitive and learning problems in Duchenne, supports the view that Duchenne muscular dystrophy is not only a muscular disorder but also a disorder affecting the brain. It is important for clinical practice to take in account this heightened association. More research is needed to examine this association and its consequences.

New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes, mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise patient mobility and quality of life, and in the most severe cases lead to complete paralysis and premature death. Although muscular dystrophies still lack an effective therapy, several novel strategies are entering or are ready to enter clinical trials. Here we review the main experimental strategies, namely drug, gene and cell therapies, outlining their goals and limitations. We also provide an update of ongoing or planned clinical trials based on these strategies.

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How Duchenne Affects the Body


– Musculoskeletal
– Heart unction
– Cognition and Learning
– Current Options for Care and Management
Coordination of Care
Corticosteroid Therapy
Musculoskeletal Management
– Physical Therapy
– Active Exercise
– Hydrotherapy
– Stretches
– Posture in Wheelchairs
Orthotics
Scoliosis and Surgery
Respiratory and Cardiac Care
– Respiratory Management
– Monitoring and Care of the Heart
Early Learning Strategies
Although we know, for some of you, this diagnosis is very new, while others have an intimate knowledge of the disease, we are here to help and continuously investigate ways to improve care for your son. We believe this CD to be an excellent source of information to help you help your son.
The CD is available now. To order your CD please email Maxine at duchennepp@mdsa.org.za
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Introduction to DMD


Muscular Dystrophy is a broad term used to label gene-related disorders that affect muscles throughout the body. There are more than 20 specific genetic disorders considered to be Muscular Dystrophy.
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Moving and Handling Children as well as adapting your home.


Two pieces of legislation affect the moving and handling of pupils in school: the Health and Safety at Work Act 1974 recommends that a moving and handling policy is developed to cover those who need help (children with special needs, for example) and those who will be doing the moving and handling; the Manual Handling Operations Regulations 1992 place a legal obligation on employers and employees to avoid lifting wherever possible, when moving and handling objects or people.
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PTC Latest News


PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control processes, today announced positive interim data from a Phase 2 clinical trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results from the first two cohorts of the three-cohort study show that treatment with PTC124 was associated with increases in muscle dystrophin expression and reductions in serum creatinine kinase values in at least 50 percent of evaluable patients. These data were presented today at the 59th American Academy of Neurology (AAN) Annual Meeting.
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“I WILL” heavy gauge wristbands


PPMD is proud to offer these heavy gauge rubber wristbands (similar in style to the CANSA bands) to help the DMD community show their strength and determination. Please note: these are the only wrist bands that benefit Duchenne muscular dystrophy South Africa directly. Wristbands are black and white with the phrase “I WILL” and the PPMD logo stamped into the rubber. And they are now available, contact Maxine to order.
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FAQ's about the use of splints and bracing.


Orthotists, the people who are specially trained in making splints, have devised a system of naming splints depending on which joints they control. AFO’s are ankle-foot orthoses and come from the toes or mid-foot to below the knee. UF doctors cast net to help muscular dystrophy patients GAINESVILLE, Fla. Patients and families affected by muscular dystrophy urgently need to take practical steps to help slow the destructive course of the disease, say University of Florida researchers who today (Sept. 2, 2005) have launched a Web-based effort to collect information from people coping with the illness.
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Are Males with Duchenne Muscular Dystrophy at Risk for Reading Disabilities?


Males with Duchenne muscular dystrophy have subaverage cognitive capacities and may manifest more specifically language-related deficits. In the current study, the information-processing capacity, reading performance, and behavioral functioning of 25 Dutch males with Duchenne muscular dystrophy (mean age 10.1 years) were systematically assessed.
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PPUK Learning and Behaviour toolkit for Duchenne Muscular Dystrophy


To all the families and boys with Duchenne Muscular Dystrophy who have inspired us all. We want this toolkit to help young men with DMD realise their potential and to lead longer and more fulfilled lives.
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Cardiorespiratoryfunction in the muscular dystrophies.


A presentation by Monique Ryan – Childrens Hospital at Westmead. First US Trial of DMD Gene Therapy Under Way COLUMBUS, Ohio, March 29, 2006 The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Childrens Hospital, the Muscular Dystrophy Association (MDA), Childrens Hospital, and Asklepios Biopharmaceutical Inc. (AskBio) announced today.
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New Prospects for treating Muscular Dystrophy


A study on mice suggests that a type of stem cells found in blood vessels may someday be able to regenerate wasting muscle in muscular dystrophy (MD) patients. Read more Cardiac MRI at CCHMC Presentation by William Gottliebson MS MD, Cardiology, CCHMC. Cardiac MRI at CCHMC Presentation by William GottliebsonMS MD, Cardiology, CCHMC. Consensus statement on the role of glucocorticoid corticosteroids in Duchenne muscular dystrophy This document reviews the evidence and, presents a protocol of assessment and management for the use of glucocorticoid corticosteroids in Duchenne Muscular Dystrophy (DMD). Steroids/Nutritional Supplements/Antibiotics There are multiple steroid/supplemental treatments for DMD although there is little agreement (even among researchers and clinicians) about many of them. Exon skipping as a potential therapy for DMD. Scientists and Clinicians from all over the world gathered in Monaco to discuss the recent advances in Exon skipping as a potential therapy for DMD.