Introduction to advanced therapies

Advanced therapies, such as gene therapy and cell therapy, are a new area of treatment development that presents unique challenges, risks and opportunies for rare disease patients and families.

Rare disease patient groups play a critical role in advocating for advanced therapies and helping their community to understand:

How advanced therapies work
The risks and benefits invovled in these therapies
How these life-changing therapies are developed, funded an accessed
And more.

Understanding the basics of genetics helps rare disease patient groups and their community make informed decisions about their care and treatment options. The same can be said for understanding the basics of advanced therapies.

Before enrolling in our advanced therapies course, we suggest enrolling in our ‘Introduction to genetics’ guide to gain the background knowledge needed to fully grasp the concepts disscussed in this advanced therapies course.

Advanced therapies are a rapidly growing treatment option, so enroll in our guide now to learn the basics of what advanced therapies can offer you and your patient group if applicable.

With thanks to:

Alice Smail, who volunteered with Beacon during Summer 2023 while getting her Masters at the University of Cambridge in genomics! Alice researched, wrote and produced the graphics of this guide for our rare disease charity on a voluntary basis.

My name is Alice and I have really enjoyed volunteering at Beacon over the summer.

I became interested in the genetics of rare disease after a close family member was diagnosed with a very rare neurodevelopmental disorder, after a long diagnostic odyssey.

I have recently finished a master’s degree in genomics, where I had the opportunity to study a group of rare conditions known as Mendelian Disorders of the Epigenetic Machinery, and in the future I hope to contribute to rare disease research!