Send us a text Imagine your body's best defenders, your immune cells, are ready for duty but locked away, unable to fight. 🛡️ For individuals with the ultra-rare WHIM syndrome, this isn't just an idea; it's the biological reality they live with every day. In this episode of March Forward, we explore WHIM syndrome, a genetic condition where a faulty signal traps vital white blood cells in the bone marrow. But this is also a story of a major breakthrough ✨. We dive into the science behind...

Imagine your body's best defenders, your immune cells, are ready for duty but locked away, unable to fight. 🛡️ For individuals with the ultra-rare WHIM syndrome, this isn't just an idea; it's the biological reality they live with every day. In this episode of March Forward, we explore WHIM syndrome, a genetic condition where a faulty signal traps vital white blood cells in the bone marrow. But this is also a story of a major breakthrough ✨. We dive into the science behind Mavorixafor, t...

What happens when standard treatments for a rare cancer like synovial sarcoma fall short? Could Empowering the answer lie in empowering your own immune system? 🛡️ In this episode of March Forward, we explore Afamitresgene Autoleucel (Tecelra), an innovative cell therapy offering a new avenue of hope for patients with advanced synovial sarcoma. This cutting-edge treatment takes a patient's own immune cells, 're-educates' them in a lab to specifically target cancer cells (targeting MAGE-A4, a p...

What if we could gently fix the tiny genetic 'typos' that cause devastating rare diseases, right where they are in the body? 🧬 In this episode of March Forward, we dive into In Vivo CRISPR Base Editing, a cutting-edge science offering a new horizon of hope ✨ for patients and families navigating rare genetic conditions. We explore how this groundbreaking technology aims to make ultra-precise changes to DNA inside the body, potentially correcting the root cause of certain diseases. Learn how ...

For families navigating Lysosomal Storage Diseases, the routine of Enzyme Replacement Therapy is a familiar path. But what if the future holds new and different options? In this episode of March Forward, we're exploring the cutting-edge science that could change how we treat these complex conditions. We're asking the big questions that many in our community think about: 🧠 Can we get treatments safely past the blood-brain barrier? 🤔 How do you weigh a proven, lifelong therapy against a one-t...