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Category: Rare Disease Research

Rare Disease Moms Make Their Voices Heard | Three Inspirational Stories

By AGT | Feb 28, 2021

Contributing Author: Gina Hagler There are 7,000 known rare diseases in the world. Each of them affects less than 1% of the global population, although ultra-rare diseases affect fewer still. The majority of rare and ultra-rare diseases are monogenic in nature, meaning they are caused by an error in a single gene. Years ago, a rare disease diagnosis left parents… [Read More]

5 Ways Gene Therapy Companies are Transforming Rare Disease Cures

By AGT | Feb 12, 2021

Contributing Author: Gina Hagler Historically, developing rare disease cures and treatments has been challenging. The small, globally dispersed patient population made it difficult and expensive to bring together enough individuals for a clinical-stage trial. The small patient population of fewer than 1% of the population also made it impossible to recoup the investment in research and development costs required for… [Read More]

3 Gene Therapy Companies Powering a Revolution in Rare Disease Cures

By AGT | Jan 20, 2021

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]

Rare Diseases: Why Are They Difficult to Cure?

By AGT | Dec 13, 2020

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]

9 Reasons Treatment of Rare Diseases Will be Big in 2025

By AGT | Oct 26, 2020

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]

Treatment of Rare Diseases: What You Need to Know

By AGT | Aug 25, 2020

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]

Rare Disease: 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

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]

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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