A Foundation Building Strength

Exciting News: Nemaline Myopathy Gene Therapy Research Advances! We're thrilled to share progress in Nemaline Myopathy research! Dr. Afrooz Rashnonejad and her team, funded by AFBS, are developing a potential gene therapy for NEM3. Their innovative approach involves silencing the faulty gene and replacing it with a healthy one. Initial results in cells and mice have been promising, and the team is now moving to the next phase of pre-clinical trials. Read more about this exciting research and its implications for the NEM3 community on our blog: https://lnkd.in/gyn5635m #nemalinemyopathy #genetherapy #research #AFBS #preclinicaltrials

𝗥𝗲𝘀𝗲𝗮𝗿𝗰𝗵 𝗕𝗿𝗶𝗲𝗳: 𝗣𝗿𝗼𝗺𝗶𝘀𝗶𝗻𝗴 𝗨𝗽𝗱𝗮𝘁𝗲𝘀 𝗶𝗻 𝗡𝗲𝗺𝗮𝗹𝗶𝗻𝗲 𝗠𝘆𝗼𝗽𝗮𝘁𝗵𝘆 𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗮𝗻𝗱 𝗗𝗿𝘂𝗴 𝗥𝗲𝗽𝘂𝗿𝗽𝗼𝘀𝗶𝗻𝗴 𝗗𝗲𝘃𝗲𝗹𝗼𝗽𝗺𝗲𝗻𝘁 Dr. Vandana Gupta and her team at Brigham & Women’s Hospital, with funding from A Foundation Building Strength (AFBS), are making exciting progress in the quest to find treatments for Nemaline Myopathy (NM). This rare neuromuscular disease affects muscle function and can significantly impact quality of life. Here’s a quick update on their recent research breakthroughs in Nemaline Myopathy gene therapy and drug repurposing. 𝙍𝙚𝙨𝙚𝙖𝙧𝙘𝙝 𝙀𝙣𝙩𝙚𝙧𝙨 𝙖 𝙉𝙚𝙬 𝙋𝙝𝙖𝙨𝙚 After successfully developing mouse & zebrafish models (crucial to understanding the disease process and testing potential therapies) of NM causing Kelch genes KLHL40, KLHL41 and KBTBD13, Dr. Gupta’s team is now working on developing gene therapy & small molecule (drug) therapy for the rare forms of NM caused by mutations in these genes. 𝘽𝙞𝙜 𝙉𝙚𝙬𝙨 𝙤𝙣 𝙩𝙝𝙚 𝙏𝙝𝙚𝙧𝙖𝙥𝙚𝙪𝙩𝙞𝙘 𝙁𝙧𝙤𝙣𝙩 Gene Therapy: Early results from AAV gene therapy in mice with severe NM have shown improvement in both lifespan and muscle function. This is a major step forward and warrants further investigation. Drug Repurposing: The team screened hundreds of FDA-approved drugs on their zebrafish model of NM. They’ve identified 16 promising drug candidates, opening the door for additional research into their potential therapeutic use. 𝙒𝙝𝙖𝙩 𝙏𝙝𝙞𝙨 𝙈𝙚𝙖𝙣𝙨 𝙛𝙤𝙧 𝙉𝙈 𝙋𝙖𝙩𝙞𝙚𝙣𝙩𝙨 These findings represent significant progress toward effective treatments for NM. While more research is needed, Dr. Gupta’s team is paving the way for a brighter future for those affected by this rare disease. AFBS is proud to support this vital work and remains committed to advancing research that improves the lives of individuals with NM. We look forward to sharing further updates as Dr. Gupta’s research progresses! #NemalineMyopathy #RareDisease #NMResearch #GeneTherapy #DrugTherapy #AFBS

𝟰𝟮 𝗿𝗲𝘀𝗲𝗮𝗿𝗰𝗵 𝘀𝘁𝘂𝗱𝗶𝗲𝘀 𝗳𝘂𝗻𝗱𝗲𝗱. 𝗖𝗼𝘂𝗻𝘁𝗹𝗲𝘀𝘀 𝗹𝗶𝘃𝗲𝘀 𝘁𝗼𝘂𝗰𝗵𝗲𝗱. At AFBS, we're funding groundbreaking research, supporting families, and building a stronger NM community. Thanks to our individual and corporate philanthropic partners, AFBS is making a real difference. 𝗪𝗶𝘁𝗵 𝘆𝗼𝘂𝗿 𝗵𝗲𝗹𝗽, 𝘄𝗲 𝗰𝗮𝗻 𝗵𝗮𝘃𝗲 𝗮𝗻 𝗲𝘃𝗲𝗻 𝗴𝗿𝗲𝗮𝘁𝗲𝗿 𝗶𝗺𝗽𝗮𝗰𝘁! Do you know someone who may want to form an individual or corporate philanthropic partnership with A Foundation Building Strength? 𝗗𝗼𝘄𝗻𝗹𝗼𝗮𝗱 𝗮𝗻𝗱 𝘀𝗵𝗮𝗿𝗲 𝗼𝘂𝗿 𝗻𝗲𝘄 𝗣𝗵𝗶𝗹𝗮𝗻𝘁𝗵𝗿𝗼𝗽𝗶𝗰 𝗣𝗮𝗿𝘁𝗻𝗲𝗿𝘀𝗵𝗶𝗽 𝗣𝗮𝗰𝗸𝗲𝘁: Help others discover how they can make a transformative impact and be part of history in the making: https://lnkd.in/guhECW3a 𝗬𝗼𝘂 𝗰𝗮𝗻 𝗵𝗲𝗹𝗽 𝗰𝗵𝗮𝗻𝗴𝗲 𝘁𝗵𝗲 𝗰𝗼𝘂𝗿𝘀𝗲 𝗼𝗳 𝗡𝗲𝗺𝗮𝗹𝗶𝗻𝗲 𝗠𝘆𝗼𝗽𝗮𝘁𝗵𝘆. #NemalineMyopathy #Philanthropy #RareDisease #NMResearch #CureNM

The AFBS Summer 2024 Update video is here, giving you an exclusive behind-the-scenes look at what we've been up to these past few months. From groundbreaking research updates to celebrating the very first Nemaline Myopathy Awareness Month, we've been working hard to make a difference for the NM community! Get ready to be inspired by the incredible progress we've made together and discover how we're getting closer to finding treatments and therapies for NM. #NemalineMyopathy #NMAwareness #RareDisease #ScientificResearch #CureNM #MedicalResearch #Nonprofit #AFBS #SummerRecap

𝗘𝘅𝗰𝗶𝘁𝗶𝗻𝗴 𝗽𝗿𝗼𝗴𝗿𝗲𝘀𝘀 𝗶𝗻 𝗡𝗲𝗺𝗮𝗹𝗶𝗻𝗲 𝗠𝘆𝗼𝗽𝗮𝘁𝗵𝘆 (𝗡𝗠) 𝗿𝗲𝘀𝗲𝗮𝗿𝗰𝗵! 🔬🧬 Dr. Vandana Gupta and her team at Brigham and Women's Hospital, supported by A Foundation Building Strength (AFBS), are making promising strides in the search for treatments for NM. After successfully developing mouse & zebrafish models of NM causing Kelch Genes (KLHL40, KLHL41 and KBTBD13), Dr. Gupta's team is now working on developing gene therapy & small molecule (drug) therapy for the rare forms of NM caused by mutations in these genes. 𝗞𝗲𝘆 𝗧𝗮𝗸𝗲𝗮𝘄𝗮𝘆𝘀: 🧬 𝗣𝗿𝗼𝗺𝗶𝘀𝗶𝗻𝗴 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆 𝗿𝗲𝘀𝘂𝗹𝘁𝘀: Early results from gene replacement therapy in mice with severe NM showed improvement in the lifespan and muscle function warranting further study. 💊 𝗗𝗿𝘂𝗴 𝗥𝗲𝗽𝘂𝗿𝗽𝗼𝘀𝗶𝗻𝗴: After testing hundreds of FDA-approved drugs on zebrafish with NM, they've identified 16 promising drug candidates, laying the groundwork for further investigation of their therapeutic potential.Dr. Vandana Gupta and her team at Brigham & Women's Hospital (@brighamdpm) , supported by A Foundation Building Strength (AFBS), are making promising strides in the search for treatments for NM. These findings bring us closer to effective treatments for NM. While more research is needed, Dr. Gupta's team is paving the way for a brighter future for those affected by this rare disease. Stay tuned for updates as Dr. Gupta's team continues their important work! #nemalinemyopathy #raredisease #research #genetherapy #drugrepurposing

🔬🧬 AFBS Drives Nemaline Myopathy Research Forward with Launch of Beggs Lab Biobanking Program 🔬🧬 A Foundation Building Strength (AFBS) is proud to announce a significant advancement in the fight against Nemaline Myopathy (NM) with the launch of the Beggs Lab NM Biobanking Program at Boston Children's Hospital, led by renowned geneticist Dr. Alan Beggs. This collaborative research initiative aims to revolutionize our understanding of NM and pave the way for novel therapeutic strategies. By expanding upon an existing tissue repository and actively engaging with the NM community, the program will create an invaluable resource for scientists and researchers worldwide. Key features of the Beggs Lab NM Biobanking Program include: - Comprehensive sample collection: Expanding the existing tissue repository to include a wide range of samples from individuals with NM. - Active community engagement: Collecting clinical information and genetic data from newly diagnosed individuals. - Accessible genetic testing: Offering free genetic testing to participants without a confirmed diagnosis. - Removing financial barriers: Ensuring participation is accessible to all individuals affected by NM. This initiative represents a significant step forward in our mission to empower patients, advance research, and ultimately improve the lives of those affected by NM. Learn more about the Beggs Lab NM Biobanking Program and how you can contribute to this vital research: https://lnkd.in/gU3rdVMG #AFBS #BeggsLab #NemalineMyopathy #Biobanking #MedicalResearch #RareDisease #NeuromuscularDisease #Genetics #BostonChildrensHospital