The PJ Nicholoff Steroid Protocol for Duchenne and Becker Muscular Dystrophy and Adrenal Suppression

Introduction Duchenne Muscular Dystrophy (DMD) is the most common and severe muscle disease presenting in childhood. It is caused by mutations in the dystrophin gene, located on the X chromosome, which causes a complete absence of dystrophin protein in muscle. Becker Muscular Dystrophy (BMD) is caused by partial absence of dystrophin; this disease is less severe and less common. As an X-linked disease, both diseases almost always affect males, though some females may be affected as well. DMD and BMD have a prevalence of 1 in 5000 males, in about a 2:1 ratio.1,2 People with Duchenne Muscular Dystrophy have progressive muscle weakness, which generally begins proximally and spreads to the legs, arms and other muscles. It is now known from a newborn screening study that the onset of disease is at birth, and probably in utero.3 Boys with DMD are born with serum creatine kinase (CK) values over 2000 U·L-1. The diagnosis is typically made between 3 and 5 years of age. Progressive muscle weakness leads to the loss of ambulation at 10 to 12 years old without treatment, though ambulation can be extended by one or two years through the use of corticosteroids. There is progressive respiratory muscle weakness, which leads to hypoventilation, respiratory insufficiency, and respiratory failure in adulthood. There is also a progressive cardiomyopathy leading to cardiac insufficiency. Prior to the use of corticosteroids, assisted ventilation and other interventions, the median life ex...
Source: PLOS Currents Muscular Dystrophy - Category: Neurology Authors: Source Type: research

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Source: Postgraduate Medicine - Category: Internal Medicine Tags: Postgrad Med Source Type: research
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Source: Journal of Applied Biomechanics - Category: Sports Medicine Tags: J Appl Biomech Source Type: research
One of the challenges faced when treating a child with genitourinary rhabdomyosarcoma is what to do when a residual mass remains after organ sparring treatment. The authors present an interesting case which exemplifies the dilemma. The answer to this question is simplified for when the residual mass is associated with pain, is growing or impairs organ function, providing a rational for excision. When this is not the case concern centers around residual disease and recurrence. Today the post-treatment finding of mature rhabdomyoblasts is not a reason for exenterative surgery, however some point bear mentioning.
Source: Urology - Category: Urology & Nephrology Authors: Source Type: research
We appreciate and thank the authors for their editorial comments. Dr. Cockett was a consummate academician and teacher, as well as a tireless leader in organized urology. During his career, he studied a wide assortment of urologic problems with space travel, including urolithiasis, spermatogenesis, and voiding function in weightless environments. He identified spermatogenic arrest in primates both in space flight and immobilization.1 Caliber and uroflow did not change in Air Force personnel studied during parabolic flights simulating weightlessness when compared to ground level voiding curves.
Source: Urology - Category: Urology & Nephrology Authors: Source Type: research
This outstanding investigation reports the real-world practice patterns for shockwave lithotripsy (SWL) across multiple practice settings in Michigan. This is a unique study that could only be accomplished by leveraging the Michigan Urological Surgery Improvement Collaborative Reducing Operative Complications from Kidney Stones registry. The authors developed a list of process and outcome measures utilizing the AUA guidelines as a framework and assessed adherence to these guidelines.
Source: Urology - Category: Urology & Nephrology Authors: Source Type: research
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Source: Urology - Category: Urology & Nephrology Authors: Source Type: research
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Source: Urology - Category: Urology & Nephrology Authors: Source Type: research
NBC News and the Commonwealth Fund recently took the health care pulse of U.S. adults, asking about their concerns regarding health care and their confidence in President Trump or the Democratic presidential candidates to help fix what’s ailing the U.S. health system. This is the first poll in a series.        
Source: The Commonwealth Fund: Publications - Category: International Medicine & Public Health Source Type: research
Ulnar-sided wrist pain is a common problem in athletes that can be challenging owing to its frequent combination of overuse in conjunction with acute injury. Repetitive pronosupination, wrist flexion and extension, as well as radial and ulnar deviation can predispose the athlete to injury of ulnar structures. Careful understanding of the sport-specific injuries as well as the underlying biomechanics are key to understanding and treating the athlete. In this article, we discuss the most frequent causes of ulnar-sided wrist pain in the athlete and focus on anatomy and pathophysiology, presentation, and diagnosis, as well as ...
Source: Clinics in Sports Medicine - Category: Sports Medicine Authors: Source Type: research
Introduction Duchenne Muscular Dystrophy (DMD) is the most common and severe muscle disease presenting in childhood. It is caused by mutations in the dystrophin gene, located on the X chromosome, which causes a complete absence of dystrophin protein in muscle. Becker Muscular Dystrophy (BMD) is caused by partial absence of dystrophin; this disease is less severe and less common. As an X-linked disease, both diseases almost always affect males, though some females may be affected as well. Duchenne and Becker have a prevalence of 1 in 5000 males, in about a 2:1 ratio.1,2 People with Duchenne Muscular Dystrophy have progress...
Source: PLOS Currents Muscular Dystrophy - Category: Neurology Authors: Source Type: research
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