Prevail Therapeutics

Learn more about genetic links to Parkinson’s ? PD GENEration is an initiative from the Parkinson's Foundation which offers genetic testing and counseling at no cost for people with Parkinson’s. ? The test, which only requires a simple blood draw, looks for changes in genes related to Parkinson’s. Knowing this information can help patients and doctors better understand the condition and may open the door to more clinical trial options. ? Learn more about PD GENEration on the Parkinson's Foundation website here:?https://bit.ly/3EJJUHJ #PDGENEration #ParkinsonsDisease #GeneticTesting #ParkinsonsAwareness #ParkinsonsFoundation #Together4PD #ABCsofPD

Advances in Gaucher Disease Clinical Research: Updates from the PROCEED trial at the 21st Annual WORLDSymposium 2025. ? At Prevail Therapeutics, we are pioneering innovative gene therapy solutions to address the root causes of rare and genetic neurodegenerative diseases. Understanding the genetic foundations of these diseases is key to developing effective therapies. Through advancements in genetic testing and research, we are unlocking new possibilities for conditions such as Parkinson’s disease, frontotemporal dementia, and Gaucher disease (GD). We are excited to share the latest updates from the PROCEED trial - a Phase 1/2 study evaluating our AAV9-based gene therapy for type 1 GD. Join us at the 21st?Annual WORLDSymposium, February 3rd?– 7th , 2025, to hear how our innovative approach is advancing clinical research in GD. GD1 and GD2 posters will be presented on Feb 5th,?3:30 – 5:30 PM PST. To learn more about the PROCEED clinical trial or our research in GD and other rare diseases, please visit our website https://bit.ly/4gjVCX6 #WORLDSymposia #WORLDSymposium2025 #GaucherDisease #PrevailTherapeutics?

From patients and caregivers to clinicians and researchers – happy holidays to everyone in the rare and genetic disease community! Check out our website https://bit.ly/4gjVCX6 to see how Prevail has been working to advance gene therapy in 2024, and the great strides we are planning to take in the New Year. #FTD, #PD, #GD #happyholidays #raredisease #geneticdisease #genetherapy #frontotemporaldementia #parkinsonsdisease #gaucherdisease

Currently, there are no disease-modifying treatments for frontotemporal dementia. Management focuses on easing symptoms, as there is no known way to slow or stop the progression of the disease. While still investigational, gene therapy is being explored as a potential approach to address the underlying causes of the condition. Prevail is committed to advancing gene therapies, and improving the lives of people living with neurodegenerative diseases. Our PROCLAIM phase 1/2 clinical study is investigating an AAV9-based gene therapy in people with FTD with the GRN genetic variant. The therapy is designed to address the underlying cause of disease by delivering a healthy copy of the GRN gene, and is intended to slow disease progression after one dose. Find out more about the trial here https://bit.ly/4gt0jhn https://bit.ly/3VwyA7y #FTD-GRN #endFTD #FrontotemporalDementia #clinicaltrials #raredisease #geneticdisease #GeneTherapy

We’re excited to participate in the 2024 Parkinson Study Group (PSG) Annual Meeting! Taking place December 5th – 8th, 2024, in Nashville, TN, this event has been a pivotal gathering for over 30 years, fostering collaboration in Parkinson’s research and education. It brings together leading experts, trainees, advocates, and stakeholders to share advancements and best practices. Learn more about the meeting: ?https://bit.ly/41gpD65 Let’s work together to shape the future of Parkinson’s care. See you there! #ParkinsonsDisease #PrevailTherapeutics #Parkinson'sFoundationCentersLeadershipConference #ParkinsonsStudyGroupAnnualConference #ParkinsonsFoundation #Together4PD #ParkinsonsAwareness #PDLeadership #CentersOfExcellence #LeadershipInAction #Advocate4Parkinsons #Connect4PD #WorkingTogether

Prevail is proud to sponsor the Parkinson's Foundation Centers Leadership Conference (CLC) in Nashville this December 4th – 6th. ? The CLC is an annual conference that brings together healthcare professionals, Parkinson's Foundation staff, advocates and industry partners whose common goal is to enhance the quality of care and achieve better outcomes for individuals living with Parkinson’s Disease (PD). ? To learn more about the work we do in Parkinson’s disease or our clinical trials, please visit our website https://bit.ly/4gjVCX6 ? #ParkinsonsDisease #PrevailTherapeutics #ParkinsonsFoundationCentersLeadershipConference #Together4PD #ParkinsonsAwareness #PDLeadership #CentersOfExcellence #LeadershipInAction #Advocate4Parkinsons #Connect4PD #WorkingTogether

There are more than 53 million unpaid caregivers in the United States. Around a quarter say they have difficulties coordinating their loved one’s care, and one fifth class their own health as “fair” to “poor”.(1) Supporting caregivers can greatly improve their well-being, and help them continue their crucial role. Here are five things you can do this National Caregivers Month to help lighten their load. 1) Give them a break by spending time with the person they are caring for, even if it is just for an hour 2) Lend a helping hand with daily tasks such as grocery shopping, cooking meals, or laundry 3) Offer emotional support. You don’t need all the answers, just be a friendly ear 4) Encourage caregivers to look after themselves and engage in self-care 5) Recognize their efforts by expressing gratitude at every opportunity Let them know you care. For more on how to support the caregivers in your life https://bit.ly/3CJMbSd #NationalCaregiversMonth #FamilyCaregiversMonth #CaregiverAppreciation #CaregiverSupport #FamilyCaregiving #CaringForLovedOnes #CaregiverRecognition #ThankACaregiver #ICare 1. NAC and AARP. Caregiving in the U.S. 2020. Available at: https://lnkd.in/gAnrbSTU Last accessed: 23 September 2024

Genetic counselors are a key part of the rare disease healthcare team. By guiding people through genetic testing and explaining the results and their implications, genetic counselors make complex medical information more accessible and help the diagnosis process feel less overwhelming. This Genetic Counselor Awareness Day, we want to say a huge “thank you” to everyone carrying out this important work – thank you for educating, supporting, and empowering people living with genetic conditions to take control and make informed decisions about their and their family’s health. Wondering if meeting with a genetic counselor is right for you? Click here for more information: https://bit.ly/4fi6chk #GeneticCounselorAwarenessDay #IAmAGeneticCounselor #geneticcounselor #genetictesting #raredisease #geneticdisease?

Next week, our very own Jim Howley, Global Patient Advocacy and Engagement Lead at Prevail Therapeutics, along with the International Gaucher Alliance and some of the world’s most influential Gaucher Disease advocates will gather to celebrate how far we have come and to plan the next leg of the journey. The biennial International Gaucher Alliance Members’ Meeting will be held in Lisbon, Portugal, on November 9th-10th. It is an opportunity for member organizations to collaborate with the IGA team, help guide the alliance’s future work – and celebrate its 30th anniversary. The IGA was founded in 1994, to help people with GD who lacked access to treatments by working with pharmaceutical companies to develop charitable access programs. Today, it is an international umbrella group that represents the interests of patients with GD and groups from around the world. Visit the alliance’s website in the coming weeks to read the key insights from the Members Meeting. https://bit.ly/4hv9O0K #IGABiennial2024 #patientadvocacy #30thanniversary #GaucherDisease #GD

Prevail was proud to attend the FDA’s Rare Disease Innovation Hub public meeting on October 16th, 2024.?? The FDA’s Rare Disease Innovation Hub is designed to engage with the rare disease community, including patient and caregiver groups, trade organizations, and scientific and academic institutions. Despite recent advances in rare disease treatments, significant work remains. The Hub's ultimate goal is to enhance patient outcomes by promoting cross-center collaboration to address common challenges and facilitate the development of rare disease products. Prevail remains committed to improving health outcomes and enhancing the quality of life for the rare disease community.? To learn more about this ground breaking initiative, please visit: https://bit.ly/48l5fC5 ?? ?#RareDisease #Innovation #Healthcare #PatientAdvocacy #Research??