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Category: Rare Disease Research

Treatment of Rare Diseases: What You Need to Know

By AGT | Aug 25, 2020

Contributing writer: Gina Hagler An emerging drug development model has the potential to reduce the cost of treatments for rare diseases. What is a Rare Disease? The definition of a rare disease varies by country, with the consensus that a rare disease or condition is one that affects far less than 1% of the population. Because each rare disease affects… [Read More]

Rare Disease 2020: One Mom’s Fight to Save Her Son, Amber Freed Raises Millions to Create a Cure. Coronavirus won’t stop her.

By AGT | Aug 5, 2020

By Contributing Writer Gina Hagler There are nearly 7,000 rare diseases affecting approximately 1% of the global population. When your child is affected with one of these rare diseases, and it is so rare it does not even have a name, what do you do? Amber Freed’s decision was to fight like a mother. She recently shared with us what… [Read More]

Benefits of FDA Orphan Drug Designation: What You Need to Know

By AGT | Apr 6, 2020

by Irene Tennant VP of Clinical Product Development and Regulatory Affairs Developing treatments for rare diseases has many challenges. These challenges are different and, in many ways, more complex than what is experienced during development of a drug intended for a larger patient population. The complexities can range from scientific, ethical and operational to financial. Due to these hurdles, the… [Read More]

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