Gene Therapy Myths And Facts PDF

Myths and Facts About Gene Therapy Gene therapies use genetic materials a lot remains unknown about this type of therapy, to treat or prevent disease. Genes...

Myths and Facts About Gene Therapy Gene therapies use genetic materials a lot remains unknown about this type of therapy, to treat or prevent disease. Genes including the long- contain the instructions needed to term risks. Patients make the proteins our bodies require. and caregivers must thoroughly understand Alteration of any gene that results in missing these unique risks or abnormal instructions may impact how and challenges before our bodies work. Gene therapy involves the deciding to pursue use of a vector as a sort of envelope to gene therapy. deliver necessary genetic materials (the The lists below contain “package”) to restore or improve the protein- facts and unproven making instructions. myths. Understanding the While gene therapy offers hope for many facts can help you make the individuals living with neuromuscular disease, most of gene therapies. Facts These statements are true: Gene therapy affects individuals differently Researchers have not determined how and can cause severe side effects or even long the effects of gene therapy treatments death in some. will last nor the extent of benefit over time. Gene therapies require months-long Factors that may impact the effectiveness monitoring and treatment of side effects after and durability of gene therapies include age, infusion of the treatment. treatment dose, and disease progression. Gene therapy is irreversible and can result in Gene therapy targets specific cells. permanent changes to the body. A person’s basic genetic composition Over the past two decades, MDA has remains unchanged; only the cells targeted invested over $125M in gene therapies for by the therapy change. many neuromuscular diseases — ranging Not everyone will be eligible for gene from basic science research to clinical trials. therapy. Considerations of age, function, MDA’s investments have led to the pre-existing antibodies, etc., may development of clinical-ready gene therapies determine eligibility. for diseases such as spinal muscular atrophy Early studies show that gene therapy may (SMA) and Duchenne muscular dystrophy slow the progression of some diseases. (DMD), with many more in the drug Receiving gene therapy may affect access development pipeline. to other types of treatment later. MDA-funded researchers conducted the Long-term effects of gene therapy remain first human gene therapy clinical trial for unknown due to the limited data available. muscular dystrophy in 1999. DISCLAIMER: This document is meant to inform and educate the community. The information presented is not intended to replace discussions with your healthcare provider and is not and should not be considered to be medical advice. Please consult with your healthcare team and/or insurance company for information specific to you. mda.org 1-833-ASK-MDA1 facebook.com/MDAOrg @MDAorg 1  Myths These statements are false: Gene therapy is a cure. Gene therapy is simple. Gene therapy can be reversed. Gene therapy is guaranteed to show beneficial results. Gene therapy treatments are not yet of proven clinical value. Gene therapy is available to treat everyone with a neuromuscular disease diagnosis. Any physician can prescribe gene therapy treatment safely. MDA’s Involvement in Gene Therapy MDA has invested over $125M in the development of gene therapy (GTx) for neuromuscular diseases over the past 20 years. With new gene therapy drug approvals in the pipeline, MDA is here to help facilitate access and provide support and education to the neuromuscular disease community. By Phone: 1-833-ASK-MDA1 (1-833-275-6321) By Email: [email protected] Gene therapy support from MDA MDA Gene Therapy Support staff are available Monday through Friday, 9 a.m. to 5 p.m. CT. Answers to inquiries can be expected within one to two business days. MDA services are available only in the US. If you live outside the US, we may be able to connect you to muscular dystrophy groups in your area. This document was prepared with input from Barry J. Byrne, MD, PhD, University of Florida; John W. Day, MD, PhD, Stanford University; Natalie Goedeker, MSN, CPNP, Washington University in St. Louis; and Julie Parsons, MD, Children’s Hospital of Colorado. DISCLAIMER: This document is meant to inform and educate the community. The information presented is not intended to replace discussions with your healthcare provider and is not and should not be considered to be medical advice. Please consult with your healthcare team and/or insurance company for information specific to you. mda.org 1-833-ASK-MDA1 66-0074 May 2023 facebook.com/MDAOrg @MDAorg 2