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Terms of Use

Welcome to the website (“Site”) of Prevail Therapeutics Inc., a wholly owned subsidiary of Eli Lilly and Company (collectively, “Prevail”). Please read and review these terms of use (“Terms of Use”) carefully. By using this Site, you acknowledge that you have read, understand and agree to be bound by the following terms and all applicable laws and regulations. If you do not agree to these Terms of Use, you are not permitted to access or use this Site.
 
In addition to these Terms of Use, you should also read and be aware of the provisions of our Privacy Statement, which discloses Prevail’s practices regarding the collection and use of your personal information, and other policies that govern other parts of our Site.
 
Prevail reserves the right to update or modify these Terms of Use at any time without prior notice. Your use of this Site following any such change constitutes your agreement to follow and be bound by the Terms of Use as changed. For this reason, we encourage you to review these Terms of Use every time you use this Site.
 
The information provided on this Site is presented in summary form, is general in nature, and is provided for informational purposes only. The information on this Site is intended only for the purpose of helping patients and family members better understand certain health conditions and treatment options. The content is not intended in any way to be a substitute for professional medical advice and should not be interpreted as treatment recommendations. Only a physician who has had an opportunity to interact with the patient in person, with access to the patient’s records and the opportunity to conduct appropriate follow-up, can provide recommendations for treatment.
 
Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Neither the content nor any other service offered by or through this Site is intended to be relied on for medical diagnosis or treatment, without a physician’s interaction and involvement. Never disregard medical advice or delay in seeking it because of something you have read on this Site.
 
THIS SITE AND THE CONTENT ARE PROVIDED “AS IS”. PREVAIL, ITS PARENT COMPANY, ITS AFFILIATES, ITS LICENSORS AND ITS SUPPLIERS TO THE FULLEST EXTENT PERMITTED BY LAW, DISCLAIM ALL WARRANTIES, EITHER EXPRESSED OR IMPLIED, STATUTORY OR OTHERWISE, INCLUDING BUT NOT LIMITED TO THE IMPLIED WARRANTIES OF MERCHANTABILITY, NON-INFRINGEMENT OF THIRD PARTIES’ RIGHTS AND FITNESS FOR PARTICULAR PURPOSE. SPECIFICALLY, PREVAIL, ITS PARENT COMPANY, ITS AFFILIATES, ITS LICENSORS AND ITS SUPPLIERS MAKE NO REPRESENTATIONS OR WARRANTIES ABOUT THE ACCURACY, RELIABILITY, COMPLETENESS, CURRENTNESS, SUITABILITY OR TIMELINESS OF THE CONTENT, SOFTWARE, TEXT, GRAPHICS, TOOLS, LINKS, PRESCRIBING INFORMATION, MEDICATION GUIDES OR OTHER COMMUNICATIONS PROVIDED ON OR THROUGH THE USE OF THIS SITE OR ANY SITE OR SITES “LINKED” TO THIS SITE. PREVAIL MAKES NO WARRANTY THAT THIS SITE WILL BE AVAILABLE, UNINTERRUPTED, ERROR FREE OR FREE OF VIRUSES OR OTHER HARMFUL COMPONENTS.
 
TO THE FULLEST EXTENT PERMITTED BY APPLICABLE LAW, IN NO EVENT SHALL PREVAIL, ITS PARENT COMPANY, ITS AFFILIATES, ITS LICENSORS, ITS SUPPLIERS OR ANY THIRD PARTIES MENTIONED ON THIS SITE BE LIABLE FOR ANY DAMAGES (INCLUDING, WITHOUT LIMITATION, INCIDENTAL AND CONSEQUENTIAL DAMAGES, PERSONAL INJURY/WRONGFUL DEATH, LOST PROFITS OR DAMAGES RESULTING FROM LOST DATA OR BUSINESS INTERRUPTION) RESULTING FROM THE USE OR INABILITY TO USE THIS SITE OR THE CONTENT OR ANY FAILURE OF PERFORMANCE, ERROR, OMISSION, INTERRUPTION, EFFECT, DELAY OR DEFECT IN OPERATION OR TRANSMISSION, COMPUTER VIRUS, LINE SYSTEM FAILURE OR LOSS OF USE RELATED TO THIS SITE OR THE CONTENT, WHETHER BASED ON WARRANTY, CONTRACT, TORT OR ANY OTHER LEGAL THEORY AND WHETHER OR NOT PREVAIL IS ADVISED OF THE POSSIBILITY OF SUCH DAMAGES. IF YOU ARE DISSATISFIED WITH THIS SITE OR THESE TERMS OF USE, YOUR SOLE AND EXCLUSIVE REMEDY IS TO DISCONTINUE USE OF THIS SITE.
 
Any claims arising in connection with your use of the Site or any content must be brought within one (1) year of the date of the event giving rise to such action occurred.
 
As a resource to our visitors, this Site provides links to other web sites. However, because we do not control the content of the other websites we may link to, and due to their constantly changing nature, we cannot be responsible for the content, practices or standards of third-party sites. Prevail does not endorse the content on any third-party websites. Prevail is not responsible for the content of linked third-party sites, sites framed within the site, or third-party advertisements, and does not make any representations regarding their content or accuracy. Your use of third-party websites is at your own risk and subject to the terms and conditions of use for such sites.
 
Everything that you read or see on the Site is copyrighted or otherwise protected and owned by Prevail or a third party who licensed or granted to Prevail the right to use such material. Unless otherwise expressly noted, nothing that you read or see on the Site may be copied or used except as provided in these Terms of Use, the Copyright Policy or with the prior written approval of Prevail.
 
Unless otherwise indicated, all logos, names, designs, and marks on the Site are trademarks or service marks owned or used under license by Prevail. The use or misuse of any of these marks or other information is strictly prohibited. Nothing contained herein shall be construed as conferring by implication, estoppel or otherwise any license or right under any patent or trademark of Prevail or any third party.
 
Except as expressly provided above nothing contained herein shall be construed as conferring any license or right under any Prevail copyright. Except as expressly set forth on the Site, if you submit any information to us, including any comments, remarks, suggestions, ideas, notes, drawings, graphics, concepts, or other information, you are giving that information, and all your rights in it, to Prevail free of charge and such information shall be deemed to be non-confidential and Prevail shall have no obligation of any kind with respect to such information and shall be free to reproduce, use, disclose and distribute the information to others without limitation, without your consent or any compensation to you or anyone else. Prevail shall be free to use any know-how or techniques contained in such information for any purpose whatsoever including but not limited to developing, manufacturing and marketing products incorporating such information. This is true whether you submit such information to us by email, through a form on the Site, on a bulletin board, or in any other manner. Prevail may from time-to-time monitor, review and, in its sole discretion, modify or delete any postings you make on the Site however, Prevail is not obligated to do so.
 
You agree not to submit or transmit any material that is unlawful, threatening, libelous, defamatory, obscene, pornographic, profane, or might in any other way violate any law, regulation, or rule. You are solely responsible for any material you submit to the Site. You further agree not to upload, email, post or transmit to, or distribute or otherwise publish through the Site any material which disrupts the normal operation of the Site, including posting or otherwise transmitting material that is not related to the subject at issue or otherwise restricts or inhibits any other user from using the Site. Through your usage of the Site, you may submit and/or Prevail may gather certain limited information about you and your web site usage. We are free to use such information for any purpose we deem appropriate, including marketing purposes.
 
To obtain full access to the Site, you may be required to complete a registration. You are responsible for maintaining the confidentiality of your account and password and for restricting access to your computer. You accept responsibility for all activities that occur under your account or password and such use shall be deemed to be use by you. You will ensure that all use of your account fully complies with these Terms of Use. Transfer of the account by you to any other person or entity is prohibited.
 
When using the Site, information will be transmitted over a medium that is beyond the control and jurisdiction of Prevail and its suppliers. Accordingly, Prevail assumes no liability for or relating to the delay, failure, interruption, or corruption of any data or other information transmitted in connection with use of this Site.
 
Prevail Therapeutics Inc. is located in New York, New York and Eli Lilly and Company is located in Indianapolis, Indiana, in the United States of America (USA). This Site is intended for the exclusive use of residents of the USA. Prevail makes no representation that the materials on the Site are appropriate or available for use in other locations. Access to the content may not be legal by certain persons or in certain countries outside of the USA. If you access this Site from outside the United States, you do so at your own risk and are responsible for compliance with the laws of your jurisdiction.
 
These Terms of Use are governed by the internal substantive laws of the State of Indiana, without resort to its conflict of laws principles. If any provision of these Terms of Use is found to be invalid by any court having competent jurisdiction, the invalidity of such provision shall not affect the validity of the remaining provisions of these Terms of Use. You expressly agree that exclusive jurisdiction for any dispute with Prevail, or in any way relating to your use of this Site, resides in the courts of the State of Indiana. Prevail may modify these Terms of Use at any time without notice to you. Prevail may terminate this agreement, terminate your access to all or part of the Site, or suspend any user’s access to all or part of the Site, at any time, without notice to you, if it believes, in its sole judgment, that you have breached or may breach any term or condition of this agreement, or for its convenience. You may terminate this agreement at any time by destroying all materials received from the Site and ceasing to use the Site.
 
Except as expressly provided in a particular “legal notice” on this Site, these Terms of Use and the documents referenced here constitute the entire agreement between you and Prevail with respect to the use of this Site, and content. Your use of this Site is also subject to the Copyright Policy and the Privacy Statement.

Definitions

β-glucocerebrosidase (GCase)

An enzyme in the body. It helps break down fatty substances. These substances are recycled inside lysosomes, which are the recycling centers of cells. If GCase doesn’t work properly or is missing, fat builds up in lysosomes. This buildup happens in places like the spleen, liver, and bone marrow. It can cause health problems, such as Gaucher disease.

Cerebrospinal fluid (CSF)

A clear fluid that surrounds and cushions the brain and spinal cord, protecting them and helping transport nutrients and waste.

Cisterna magna

An area at the base of the skull where the brain and the spinal cord meet. This space is filled with cerebrospinal fluid and can be used by doctors to deliver treatments directly to the brain and spinal cord.

Clinical trials

Research studies that test new treatments or procedures in people to make sure they are safe and work well.

Dopamine transporter single-photon emission computed tomography (DaT-SPECT)

A scan that is used to diagnose neurodegenerative Parkinson’s disease from other types of Parkinson’s disease. For example, Parkinson’s disease caused by medicines or reduced blood supply to the brain.

Electrocardiogram (ECG)

A test that checks the heart’s rhythm and electrical activity using small sensors attached to the skin.

Enzyme replacement therapy (ERT)

A treatment that gives the body enzymes it’s missing or that aren’t working properly. For Gaucher disease, ERT provides a replacement for the GCase enzyme through an infusion. This helps reduce the buildup of fat in the body and manage symptoms.

Frontotemporal dementia (FTD)

A type of dementia that gets worse over time. It damages the brain’s frontal and temporal lobes. These areas control personality, behavior, language, and movement, which means FTD can lead to significant changes in how a person acts, communicates, and moves.

FTD with GRN genetic variants

FTD is a type of dementia that damages the brain’s frontal and temporal lobes, causing changes in personality, behavior, language, and movement.

About 5–10% of FTD cases are linked to genetic variants in the GRN gene. The GRN gene provides instructions for making progranulin, a protein needed to keep brain cells healthy. Genetic variants in GRN lead to lower levels of progranulin, causing brain cell damage and faster disease progression. People with these genetic variants often experience more severe and rapidly worsening symptoms.

GBA1 genetic variant

The GBA1 gene gives cells the instructions to make the GCase enzyme. This enzyme helps break down fatty substances inside lysosomes, which are the cell’s recycling centers. A GBA1 genetic variant is a change in this gene. This change can stop the GCase enzyme from working properly. When this happens, fatty substances build up in the body. This buildup can cause Gaucher disease or increase the risk of Parkinson’s disease.

Gaucher disease (GD)

A rare condition passed down through families, caused by changes (genetic variants) in the GBA1 gene. These changes affect a specific enzyme called GCase, which is needed to break down certain fatty substances. When GCase doesn’t work properly, these substances build up in lysosomes, the recycling centers of cells. Over time, this buildup can affect organs like the spleen, liver, and bone marrow, causing symptoms such as fatigue, bone pain, and organ swelling.

Gene therapy

A type of treatment that uses genetic material, such as DNA or RNA, to fix or replace faulty genes in the body. This treats or prevents certain diseases by enabling the genes to work as they should.

Genetic variant

A genetic variant is a change in a gene that can affect how it works. Changes in genes are mostly harmless. But, in some cases, they can make the body work differently and increase the risk of certain diseases. Genetic variants were previously called ‘mutations’.

Immunosuppressants

Medicines that lower the activity of the immune system. In gene therapy, they are used to reduce the chances of the immune system reacting to the inactivated virus that delivers the gene therapy to cells.

Informed Consent Form (ICF)

A document that provides important details about a clinical trial. It explains the potential risks and what is required before, during, and after the clinical trial. It also outlines the rights of participants. Participants sign the form to show they understand the clinical trial and agree to take part.

Infusion

A way of delivering fluids, including medicine, directly into the bloodstream using a needle or small tube.

Intravenous / intravenously (IV)

A method of giving medicine or fluids directly into a vein.

Lumbar puncture

A medical procedure where a small sample of cerebrospinal fluid is taken from the lower back using a needle. It’s sometimes called a spinal tap.

Lysosomes

Small parts of cells that act as recycling centers. They break down and recycle waste materials, including fatty substances, to keep the cells healthy. When lysosomes don’t work properly, these substances can build up and cause health problems, such as in Gaucher disease.

Magnetic resonance imaging (MRI)

A medical scan that uses powerful magnets and radio waves to create detailed images of the inside of the body, like the brain or spine.

Parkinson’s disease (PD)

A brain condition that affects movement. It happens when certain brain cells that produce a chemical called dopamine stop working properly. Without enough dopamine, the brain cannot send proper signals to the body. This can cause symptoms like tremors, stiffness, slow movements, and trouble with balance.

Parkinson’s disease with GBA1 genetic variants

Parkinson’s disease is a brain condition that affects movement, caused by the loss of brain cells that produce dopamine. Without enough dopamine, symptoms like tremors, stiffness, and slow movements can occur.

Some cases of Parkinson’s are linked to genetic variants in a gene called GBA1, the most common genetic risk factor for the condition. These genetic variants increase the risk of developing Parkinson’s and can lead to earlier and faster-progressing symptoms.

Peripheral vein

A vein found in areas like the arms, hands, legs, or feet. These veins are closer to the surface of the body and are commonly used for giving medications or drawing blood.

Placebo-controlled

In a clinical trial where there are two groups of participants, one group receives the active treatment or intervention, while the other group receives an inactive treatment or intervention (the placebo). The placebo is administered in the same way as the active treatment but does not contain any medicine.

Small interfering RNA (siRNA)

A type of therapy that uses small pieces of RNA, a natural molecule in cells, to block specific genes from making harmful proteins. siRNA works by ‘silencing’ genes that may cause or worsen certain diseases. This therapy is different from traditional gene therapy, as it focuses on turning off harmful gene activity rather than replacing or fixing genes.

Substrate reduction therapy (SRT)

An oral treatment (tablet) used for Gaucher disease. It works by partially stopping the body from making fatty substances. These substances are usually broken down by an enzyme called GCase. When GCase doesn’t work properly, these substances build up in lysosomes, the recycling centers of cells. SRT helps reduce this buildup and manage symptoms caused by Gaucher disease, such as organ swelling, fatigue, and bone pain.

Type 1 Gaucher disease

Type 1 Gaucher disease is the most common form of Gaucher disease, a rare inherited condition caused by genetic variants in the GBA1 gene. These genetic variants affect an enzyme called GCase, leading to a buildup of fatty substances in lysosomes, the recycling centers of cells.

Type 1 Gaucher disease usually begins during adolescence but can start at any age. Symptoms include bone pain, fractures, enlarged organs, bruising, and fatigue. Unlike other types, Type 1 does not typically affect the brain or spinal cord.
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