Research Learning Hub
We have collated some information and explainer videos to help our friends and supporters better understand the science behind the research. We have also included some information about the processes required to get a medicine from bench to bedside.
We hope you will find these useful. If you find any other resources online which you think might be helpful, please do let us know so we can add them to the Hub.
Genetics 101 by National Geographic
This video provides an introduction to genetics. It explains that genes, composed of DNA within chromosomes, make up the human genome, and highlights the significance of the Human Genome Project in mapping the human genetic sequence, while also acknowledging the future opportunities and ethical considerations in genetic testing and manipulation.
Watch: Here
Beacon Resources Hub
The Hub brings together resources from across Beacon’s training programme to provide patient groups with one, central place to further their learning on topics of interest in the field of genetics and living with a rare condition.
The e-learning resources are 100% free to access, but you do need to enroll with them.
Visit their Hub here: Get Started
Some courses which may be of particular interest:
Introduction to Genetics
Medical Research Explained
Rare Disease Research and Trial Design
Drug Repurposing Explained
Navigating Health Technology Assessments
Understanding Advanced Therapies
Genetics Basics in the UK
This webpage from Gene People explains genetics in a simple way, for a UK audience. It explains how tiny instructions inside us, called genes, guide how we grow and what we’re like. It explains about chromosomes and DNA, which are like the special books where these genes are stored. There are also links to other websites and resources.
It’s a guide to understanding the amazing code that makes each of us unique – and a great place to start if you are baffled by genetics!
Learn more: Beginners Guide
The Drug Development Journey for FOP: How Medicines Become Treatments
The IFOPA have produced a video which outlines the process of developing drugs for conditions like FOP, starting with lab research to identify potential medicines. It illustrates how medicines then undergo multi-phase clinical trials to assess their effectiveness and safety in people before going to regulatory bodies to evaluate the medicine for approval.
Watch now: The Drug Development Journey
Intro to Health Technology Assessments by Beacon
This video explains Health Technology Assessments (HTA) and their application, particularly in the context of rare diseases. It highlights the concept of opportunity cost in healthcare and how HTA aims to ensure fair and consistent decisions on spending by balancing costs against patient benefits. The video also discusses the challenges of applying HTA to rare diseases due to limited information and high costs, emphasizing the growing importance of incorporating the patient voice in such evaluations.
Watch: Here
Introduction to Gene Therapy in FOP (2020)
The IFOPA hosted a webinar, led by Adam Sherman and Betsy Bogard to give an introductory overview of gene therapy, specifically in the context of FOP. It explains how gene therapy aims to correct mutations by adding or changing genetic material to produce normal proteins, using various approaches like gene silencing, addition, replacement, and editing. The video also discusses the challenges and considerations for FOP gene therapy, including safety, targeting, durability, and the difficulty of targeting muscle tissue.
Watch now: Introduction
Gene Therapy: Your Questions Answered
This video addresses common questions about gene therapy, covering its recent developments, effectiveness, treatment methods, and potential side effects, as compiled by the National Organization for Rare Disorders (NORD). It also clarifies the distinction between gene therapy and gene editing, and advises on how to find clinical trials.
Watch here: Gene Therapy
