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Free CME

This CME program will provide endocrinologists and other health care professionals with the knowledge about the pathophysiology of acromegaly, the need for multidisciplinary care, and when and how to initiate appropriate treatment. The activity will also provide the latest information about emerging therapies that may preserve pituitary function, improve patient outcomes, quality of life, and adherence to therapy.

Click here to participate in this free CME video series.

Pediatric Health

The prevalence of childhood disability has continued to increase, according to an article published by CBS News. A study, published in Pediatrics, reports that overall childhood disability jumped by 15.6 percent from 2001-2011, with children living in poverty experiencing the highest rates. However, children from wealthy households had the highest increase of cases, with 28.4 percent growth. In terms of type of disability, mental health and developmental disabilities saw a huge increase of 20.9 percent, while physical cases actually declined by almost 12 percent. This decline could be due to a fall in asthma-related problems, and improved child safety in cars and on bikes.

FDA Approvals & Production

A new drug from Sanofi for Gaucher disease received FDA approval yesterday, according to an article published by Bloomberg News. The drug, called Cerdelga, combats the inherited metabolic disorder by decreasing the amount of waste that is not broken down in Gaucher disease patients. This treatment comes in pill form, in contrast to the company’s 20-year-old Cerezyme, which is administered intravenously and replaces a missing enzyme that breaks down the same waste reduced by Cerdelga. Sanofi plans to price the drug similarly to Cerezyme for a year’s supply, which is around $300,000.

The Ebola Outbreak

The Nigerian Health Minister Onyebuchi Chukwu has announced that Nigeria is equipped to contain the Ebola outbreak and could likely become Ebola-free within the next week, reports Bloomberg. Currently, only two Ebola patients are in Nigeria and remain in isolation. This outbreak’s death toll has risen to over 1,200 individuals, and no known or acknowledged cure has been found.

Read the article published by Bloomberg.

Genetic Disease Treatment

A drug developed by Amicus Therapeutics, Inc. has shown to be as effective as enzyme replacement therapy in patients with Fabry disease, according to an article published by Reuters. The drug, called migalastat HCl, recently finished a second late-stage trial, and works by binding to and aiding the alpha-Gal enzyme, which is deficient in Fabry disease patients. The deficiency causes an accumulation of a specific type of fat if left untreated, which can lead to kidney failure, heart attack, or stroke. The drug would be administered orally, which is an improvement from the current bi-weekly infusions required for enzyme replacement.