Biomedical Advanced Research and Development Authority (BARDA)’s Post

For #WorldSanfilippoAwarenessDay tomorrow, we recognize the importance of spreading awareness and sparking conversations globally about #SanfilippoSyndrome, including what it is and how it impacts families. This year, one of the ways that the Cure Sanfilippo Foundation is helping to spread awareness is through its #HandsofHope viral challenge.   At JCR Pharmaceuticals, we maximize our cutting-edge J-Brain Cargo® technology to develop treatment options for devastating diseases like #SanfilippoSyndrome, and we hold out our hands to stand with the Sanfilippo community to honor the children around the world living with #SanfilippoSyndrome. Our knowledge about #SanfilippoSyndrome has benefited greatly from our conversations with the community and with the Cure Sanfilippo Foundation.   You can learn more how you can participate in the #WorldSanfilippoAwarenessDay here: https://bit.ly/4hi11PQ.   #WorldSanfilippoAwarenessDay #CureSanfilippo #HandsOfHope #SanfilippoSyndrome #RareDisease #JCRPharma #LysosomalStorageDisorders #BloodBrainBarrier #MPSIII

📢 Last week the FDA updated its progress on the establishment of the Rare Disease Innovation Hub, a cross-center initiative aimed at accelerating rare disease treatment by fostering collaboration across the agency. Community feedback was gathered at the 'Advancing Rare Disease Therapies' event last month and is being used to inform the Hub’s direction. Amy Comstock Rick, J.D., was announced as the Director of Strategic Coalitions. With collaboration across CBER, CDER, and other FDA centers, the Hub will drive regulatory science for rare diseases through a united approach. Over 7,000 rare diseases affect more than 30 million people in the United States. Since the Orphan Drug Act came into effect in 1983, the FDA has approved hundreds of drugs for rare diseases. However, many rare conditions remain life-threatening, and most still lack treatments. Read the announcement from the FDA here: https://lnkd.in/gHyX-jUx #RareDiseaseHub #FDAInnovation #RareDisease

📣 Exciting news from Rallybio! Today at #ASBMR2024 with our partners Exscientia and Millán Lab, we debuted promising nonclinical data supporting ENPP1 inhibition as a therapeutic approach for hypophosphatasia (HPP) for the first time. Thanks to Dr. José Luis Millán for presenting these findings - With the significant unmet patient need, especially among adults, our findings bring hope for a potential new treatment that could improve the lives of those battling this devastating rare disease.     We're encouraged to see this data as we continue to work with Exscientia on an ENPP1 inhibitor with improved properties compared with REV101 as a differentiated therapy to address the unmet need in patients with HPP. We expect to have more to share on our inhibitor development candidate later this year - for now, download our poster here to take a closer look: https://lnkd.in/eesRGgsr   #RareDisease #HPP #ASBMR2024 

Khondrion and Amsterdam UMC join forces to combat persistent post-COVID symptoms Khondrion Jan Smeitink Amsterdam UMC Phase 2 trial on sonlicromanol to test its ability in improving patient's symptoms & quality of life #coronavirus #COVID19 #mitochondria https://lnkd.in/eyh77gBf

🌟 FDA’s STAR Pilot Program: Accelerating Rare Disease Therapies 🌟 Exciting news for the rare disease community! The FDA has launched the *Support for Therapeutic Advancement and Research (STAR) Pilot Program*, an initiative designed to speed up the development of therapies for rare diseases. STAR aims to foster collaboration, support innovative trial designs, and provide early, ongoing feedback to developers of rare disease treatments. By streamlining the path from research to regulatory approval, the program hopes to deliver more effective, timely solutions to patients with critical, unmet needs. Let’s celebrate this step forward in making transformative therapies more accessible to rare disease patients! #FDA #RareDiseases #STARProgram #DrugDevelopment #PatientAdvocacy #HealthcareInnovation

This year, #RareDiseaseDay is on Friday, February 28. #RareDiseaseDay is the globally coordinated movement on rare diseases, working toward equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. This year’s campaign theme is “More Than You Can Imagine.” It shines a spotlight on the power of resilience and connection among the rare disease community. At JCR Pharmaceuticals, we are dedicated to developing therapies for people living with rare diseases, beginning with lysosomal storage disorders. Learn more about #RareDiseaseDay and how you can participate in raising awareness for #RareDiseases: https://bit.ly/3WpMJE5. #RareDiseaseDay #RareDisease #JCRPharma

A look back. At the World Orphan Drug Congress Europe, we ran the latest in our series of workshops to support better patient outcomes by elaborating on the needs of the different stakeholders in the healthcare ecosystem. Our CEO 💭 Christopher Rudolf explains in this video how these feed directly into Volv Global SA's development of solutions for the development and launch of new therapies, especially in areas of high unmet needs. #healthcareecosystem #unmetneeds #aiml #machinelearning #volvglobal #christopherrudolf #wodceurope2024

We are excited to announce our latest partnership with n-Lorem Foundation to develop personalized antisense oligonucleotide (ASO) treatments for patients with ultra-rare and nano-rare diseases. In many cases, nano-rare patients will succumb to their disease without ever having hope of a treatment that will halt their progressive diseases. But working with n-Lorem, we hope to change this. We will provide amidites, compounds essential to developing ASO medicines, to n-Lorem to help offset the costs of developing ASO medicines for up to 20 nano-rare patients. Learn more from Wei Jiang and Stanley Crooke about this exciting partnership below. 👇 https://loom.ly/zDaQhRk #RareDiseases #DrugDevelopment #Oligonucleotides

With over 7,000 rare diseases affecting approximately 300 million people globally, the collective burden of these conditions is profound. When it comes to rare disease clinical development, Perspectives covers a wide range of topics. Here are 2024’s top posts: Orphan Drug Designation for Rare Diseases https://hubs.ly/Q030pVy00 Understanding Market Exclusivity for Orphan Drug Products https://hubs.ly/Q030pVJ70 Quantitative Strategies for Rare Disease Clinical Trials https://hubs.ly/Q030pVzL0 Sample Size Re-Estimation for Rare Disease Clinical Trials https://hubs.ly/Q030pVPL0 Thank you to Natalia Mühlemann, Boaz Adler, Valeria Mazzanti, and the Strategic Consulting team for sharing your insights!

The small and diverse patient populations of rare diseases can introduce challenges for drug development and market access. So how can the, often limited, evidence be used to demonstrate the value of rare disease therapies? YHEC's Stuart Mealing is participating in a panel titled “Optimising Value Demonstration & Evidence in Rare Disease” at next week's World Orphan Drug Congress Europe 2024. Come along to the panel to hear insights from Stuart and the other experts on how access bodies consider value in rare disease therapies. To find out more about YHEC's work in rare diseases or to get in touch with Stuart, please contact us at: yhec@york.ac.uk #WODC #WorldOrphanDrugCongress #OrphanDrug #RareDisease