Prevail Therapeutics

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??

Prevail congratulates Aparito - a wholly owned subsidiary of Eli Lilly and Company, on celebrating its 10-year anniversary. This marks a decade of pioneering advancements in healthcare technology. Aparito digitizes clinical trials and accelerates drug development for patients with life-limiting diseases by supporting patient-centric clinical trials. Here’s?to?another?decade?of?growth,?innovation,?and?impactful?change!? To learn more about Aparito, please visit: https://bit.ly/3AiCcT5 #Aparito?#EliLilly?#DigitalHealth?#HealthTech?#RareDisease #Innovation

We can’t wait to share the latest information on our investigational gene therapy for Gaucher Disease at ESCGT 2024 in Rome! ? Sarah Neuhaus, a senior medical director at Prevail, will talk about the PROCEED trial, a Phase 1/2 dose-finding study that is evaluating our AAV9-based therapy for the peripheral manifestations of type 1 GD. Olga Uspenskaya, Prevail Therapeutics' Vice President of Clinical Development?will also have a poster, looking at data from the PROCLAIM trial for frontotemporal dementia, on display during the meeting. ? Follow the link for more information https://bit.ly/4eSPBQS ? #ESCGT #ESCGT2024 #rarediseases #genetherapy #gaucherdisease #GD

Could you be a part of the next breakthrough? At Prevail Therapeutics, we are passionate about advancing the conversation on genetic testing for rare diseases. Genetic testing involves analyzing a person’s DNA to identify changes or mutations in their genes or chromosomes, which can help in understanding and managing various conditions. Genetic testing can identify many disorders, including?frontotemporal dementia (FTD).?FTD is caused by mutations in the GRN gene in about 5–10% of cases. We are excited to highlight the Prevail and Sano Genetics Program that you can participate in from the comfort of your home. This study aims to better understand the genetics of FTD, empowering researchers to potentially identify treatment options. Free at-home genetic testing and counselling before and after your test are provided. To learn more and enroll visit: https://bit.ly/4f3y9ca Together, we can make a difference in the lives of those affected by FTD. #GeneticTesting #NeurodegenerativeDiseases?#FrontotemporalDementia #PrevailTherapeutics #Research #Breakthroughs #AtHomeTesting #GeneticCounseling

Today is International Gaucher Day! It is the perfect opportunity for all of us in the Gaucher Disease community to raise awareness of this rare condition. This International Gaucher Day we are also sending our warmest birthday wishes to the International Gaucher Alliance, which has now been amplifying the voices of people living with GD for 30 years. To mark the occasion, supporters from all over the world have filmed themselves talking about how working with the patient advocacy group has helped them to manage their health and support each other. Hear their stories and discover ways you can support International Gaucher Day here https://bit.ly/4eC6uPy #GaucherDisease #PatientAdvocacy #PatientAdvocacyGroup #rarediseases #IGD #InternationalGaucherDay #GD

The International Congress of Parkinson and Movement Disorders (MDS) is committed to tackling unmet medical need – and, at Prevail Therapeutics, so are we. This year’s MDS theme is 'Movement Disorders in the Digital Age: Harnessing AI for Better Lives’. It will bring some of the world’s leading movement disorder clinicians, scientists, and healthcare professionals together to share groundbreaking research and multidisciplinary approaches to care and therapeutics. We are so excited to be part of it. PROPEL, our phase I/II clinical trial in Parkinson's Disease with GBA1 mutations (PD-GBA), has recently started enrolling for Cohort 2. Our candidate is a potentially disease-modifying, single-dose gene therapy. If you are attending MDS, please reach out. We look forward to sharing how we plan to drive innovation in gene therapy, and advance treatment options for people living with neurodegenerative and rare diseases. If you would like more information on the PROPEL trial, please visit our website https://bit.ly/3LB3Rkm #MDSCongress #MDSM #ParkinsonsDisease #GeneTherapy #NeurodegenerativeDisorders #PrevailTherapeutics #HealthcareInnovation

It’s time for the International Congress of Parkinson and Movement Disorders (MDS) – where Prevail Therapeutics will share data on our work to develop an RNA therapy with the potential to slow or even halt neurodegeneration and progression in Sporadic Parkinson’s disease (sPD). Our investigational, small interfering RNA (siRNA) therapy has recently entered a Phase I clinical trial. It has been designed to target α-synuclein mRNA and reduce α-synuclein protein aggregation. PROSPECT, a first-in-human single and multiple ascending dose trial, will evaluate its safety, tolerability, and PK/PD. With current PD treatments being symptomatic and providing diminishing benefit and increasing side effects over time, disease-modifying therapies are desperately needed. If you are attending MDS, you can visit us at our poster 762 on Sunday September 29 from 1-3pm and speak to a member of our clinical team. #MDSCongress #MDSM #ParkinsonsDisease #GeneTherapy #siRNA #NeurodegenerativeDisorders #PrevailTherapeutics #HealthcareInnovation