Zevra Therapeutics’ Post

**Study update: Sanofi's AMETHIST trial for GM2 gangliosidosis discontinued** Today it was announced by Sanofi that the AMETHIST phase 3 study of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints. This is incredibly disappointing news as we hoped it would have potentially provided a much needed treatment option for our community. We are working with our advocacy partners Cure & Action for Tay-Sachs (CATS) Foundation and the Cure GM1 Foundation to explore next steps and we will provide more details in the coming weeks when it is available. The full press release can be read here: http://bit.ly/44imRfX. Please know that this disappointment will not stop the work we are all doing to support our communities and we will continue to explore potential future treatments. If you have any questions please reach out to us. #dontgiveup #wewontgiveup #resiliency #community #advocacy #taysachs #sandhoff #gm1 #gm2 Daniel Lewi Kathleen Flynn Diana Jussila Christine Waggoner

We work with pharmaceutical companies and patient advocacy organizations to design custom, innovative programs for the patient community. We think outside of the box to create a program that is tailored towards your goals and your community. Whether you're looking for an in-person or virtual workshop, an online course, an advisory board, or something else, let's dream it up and make it happen. #patientadvocacy #advocacymatters #chronicillness

"𝘜𝘯𝘥𝘦𝘳𝘴𝘵𝘢𝘯𝘥𝘪𝘯𝘨 𝘵𝘩𝘦 𝘵𝘳𝘶𝘦 𝘪𝘮𝘱𝘢𝘤𝘵 𝘰𝘧 𝘺𝘰𝘶𝘳 𝘢𝘥𝘷𝘰𝘤𝘢𝘤𝘺 𝘦𝘧𝘧𝘰𝘳𝘵𝘴 𝘤𝘢𝘯 𝘧𝘦𝘦𝘭 𝘭𝘪𝘬𝘦 𝘯𝘢𝘷𝘪𝘨𝘢𝘵𝘪𝘯𝘨 𝘢 𝘮𝘢𝘻𝘦." Advocacy is crucial for improving patient care and reducing administrative burdens. Dr. Persing, from the University of South Alabama’s Mitchell Cancer Institute, emphasized the importance of engaging at the state level, where changes are more attainable. Here are two takeaways from his insights shared at the ASCO Annual Meeting ⬇ - 𝐏𝐞𝐫𝐬𝐨𝐧𝐚𝐥 𝐒𝐭𝐨𝐫𝐢𝐞𝐬 𝐌𝐚𝐭𝐭𝐞𝐫: Clinicians should leverage their expertise and personal stories to resonate with legislators. - 𝐄𝐧𝐠𝐚𝐠𝐞 𝐋𝐨𝐜𝐚𝐥𝐥𝐲: Meeting with lawmakers in their home states or districts can lead to more meaningful interactions. As Dr. Persing stated, "𝘋𝘰𝘯’𝘵 𝘣𝘦 𝘢𝘧𝘳𝘢𝘪𝘥 𝘵𝘰 𝘨𝘦𝘵 𝘪𝘯𝘷𝘰𝘭𝘷𝘦𝘥." Let’s continue to ensure patients are heard and our advocacy efforts are maximizing their impact. #PatientAdvocacy #PatientCare #ASCO #HealthcareProfessionals #HealthcareAdvocacy #HealthcareInnovation #PatientCenteredCare

JCR Pharmaceuticals recognizes the importance of the GM2 Voice of the Patient report, which marks a milestone for the #TaySachs and #Sandhoff patient communities. This report offers great insights from the Externally-Led Patient-Focused Drug Development meeting with the FDA. The collaboration and continued conversation will be instrumental in increasing awareness of #GM2 gangliosidosis and better understanding these devastating diseases. We support the National Tay-Sachs & Allied Diseases Association and the entire GM2 community! Read the full report here: www.ntsad.org/gm2pfdd. #GM2 #TaySachs #Sandhoff #RareDisease #BloodBrainBarrier #LysosomalStorageDisorders

Learn more about the importance of ASGCT's evidence-based #advocacy with Diane Berry, PhD, who is the Executive Vice President and Chief Global Policy & Advocacy Officer for Sarepta Therapeutics. Want to get involved? We invite you to attend the 2024 Policy Summit (link in first comment), taking place Sept. 23-24 in DC and virtually. In this 1.5 day deep dive, you'll explore: - Global regulatory strategies and convergence, featuring first-hand learnings from international regulatory agencies   - The investment landscape for #CGTs, including perspectives from venture capital and pharmaceutical companies - Emerging opportunities in #translationalscience and #personalizedmedicine   - Critical updates on #paymentpolicies, value-based arrangements, and Medicaid coverage for CGTs   - Hot topics in #clinicaldevelopment, including risk-benefit analysis, surrogate endpoints, and real-world evidence for CGTs Join us! Early registration rates end on August 23 and ASGCT's housing block closes August 19. We look forward to seeing you in DC or virtually. #ASGCTAdvocacy

Do you, your loved one, or you patient have myelofibrosis? New clinical trial opportunity 👇 From our unique position at the intersection of 3 key stakeholders — patients, researchers/clinicians, and the biopharmaceutical industry — MPN Research Foundation (MPNRF) facilitates a collaborative approach to research that elevates the patient voice in research and drug development, brings disease experts together, and accelerates industry’s understanding of an extremely complex group of diseases. An opportunity to participate in this research study: Your myelofibrosis (MF) journey can (anonymously) help researchers learn more about the treatment experience of MF patients, including their experience with healthcare providers, satisfaction with treatments, and treatment costs.  Who can participate? You may qualify to participate if you: • are 18 years of age or older • have been diagnosed with myelofibrosis • are taking medication for myelofibrosis • live in the United States What would I have to do to participate?  • Complete the qualifying questionnaire below (~5 min. time commitment) • Discuss your MF treatment experience with Magnolia Innovation over a 60-minute phone/Zoom interview; audio only Your participation in the interview would be anonymous, and you would receive $125 in compensation for your time. Magnolia Innovation is conducting this research project on behalf of a pharmaceutical company. To avoid potential bias in responses, the name of the sponsoring company will be disclosed at the end of the interview. The sponsoring pharmaceutical company will use the research findings to improve their products and services. If you are interested in participating, please click the link below to answer a few simple questions about your MF treatment experience. [Magnolia MF Patient Experience Study - https://lnkd.in/gZ5RgYag ] Be the first to know about what new clinical trials are out there by joining out community [

In the intricate landscape of MPN research and development, the patient voice is not just valuable — it's indispensable. Sharing personal journeys empowers those affected by MPNs by fostering a sense of community. At the same time, it equips researchers, clinicians, and biopharmaceutical professionals with the nuanced insights necessary to tailor discovery and development efforts to meet genuine patient needs.  The era of underrepresentation of MPN patient voices is at an endpoint. We’re marking the dawn of a collaborative, insight-driven approach in our field. Stories like those from Ignacio and countless others are pivotal, enriching our collective endeavors and driving innovation in therapeutic solutions and treatment methodologies.  Join MPN Research Foundation in championing this collaborative ethos. Together, we can enhance the synergy between patient experiences and scientific expertise, accelerating our understanding and treatment of MPNs.   Stay informed and engaged with the latest advancements and insights in MPN research by joining our community today:

In the ever-evolving landscape of #myelofibrosisresearch, a recent study published in #eJHaem, drawing on data from the phase III SIMPLIFY-1 trial (sponsored by Sierra Oncology LLC, a GSK company), brings critical insights into treatment strategies.  This analysis scrutinized the effects of #momelotinib and #ruxolitinib on bone marrow fibrosis grades—a marker of abnormality in marrow cells. While both drugs showed improvements in fibrosis grades, this did not equate to tangible benefits in patient survival or overall well-being.  Interestingly, momelotinib sustained its efficacy in managing anemia regardless of changes in fibrosis, challenging current metrics for evaluating treatment success in myelofibrosis.  Such findings, spearheaded by Stephen Oh and his team, suggest a pivot in how we measure therapeutic responses, emphasizing the need for a collaborative approach in our journey towards better outcomes.  🔍 Dive deeper into these findings and their implications for future research:  Summary: https://shorturl.at/gsJP3 Full Study: https://lnkd.in/gVn559MB Let’s collaborate to answer the pressing questions that remain.  More questions mean more urgent need to find answers. We can’t get to those solutions without you.   Be a part of the solution by joining out community today:

Building the standard. What does good look like in patient advocacy and how can we support teams with measurements and metric that matter to underserved communities. #healthequity #healthdisparities #patientadvocacy #patientcenteredcare #pharmaceuticalindustry #patientoutcomes

Interesting take on the state of #patientadvocacy How do you reconcile that the incentives for drug development are mainly monetary while the advocates (in the US) are highly mission-driven? The author advocates for more pharma-advocacy collaboration, and I'd add that we need a more sophisticated way to talk about impact of a medication than basic endpoints. The needs are clear - we need: 1️⃣ More accepted data forms - PROs / RWE are a good start - not just traditional trials and anecdotal evidence 2️⃣ A better collection mechanism - i.e., a more meaningful way to capture the patient experience without burden - not just diaries, but wearables and other innovative monitoring sensors 3️⃣ An accepted paradigm on how to evaluate; how do you weigh the potential impact of hope a new treatment might work with the potential risk and cost a patient without options would face? 4️⃣ A tool to connect the dots - i.e., what underlying connections are we not seeing? "When we make decisions that lack the necessary scientific rigor to establish robust conclusions, nobody wins." #raredisease #clinicaltrials #hope #AI #patientreportedoutcomes #RWE #alzheimers