Contributing Author: Gina Hagler Gene therapy companies are powering a revolution in rare disease cures by offering treatments that target the genetic cause of the disease. Rather than try different combinations of chemicals to create a small-molecule treatment as is the case with traditional pharmaceuticals, gene therapy uses genetic material in drug development to repair or replace the genetic cause…. [Read More]
Contributing Author: Gina Hagler Approximately 7,000 rare diseases affect 350-500 million people worldwide. The majority of these (80%) are monogenic, so-called because these genetic disorders are caused by a mutation in a single gene. While gene therapy holds great promise for the treatment of monogenic diseases in the future, efforts to find cures to date with traditional pharmaceuticals have resulted in… [Read More]
Of the 7,000 known rare diseases affecting 350 to 400 million people worldwide, most have a genetic basis and it often comes down to a single gene that is wreaking havoc and harming the person’s health. Until recently, it was impossible to replace that one gene and save the patient. Much work has been done to map the human genome… [Read More]
Contributing writer: Gina Hagler 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 so few people, the traditional drug development model has proven to be inadequate when applied to them…. [Read More]
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 that means for her and… [Read More]
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]
Subscribe to our newsletter to keep up to date with all the latest news about AGT!