March Forward

What if we told you that AI is helping us find new uses for existing medicines, bringing us closer to treatments for rare diseases? 🔍 🔬 💡In this episode of March Forward, we gently explore how Artificial Intelligence is transforming drug repurposing, offering a new horizon of hope for patients and families navigating rare conditions. We discuss how AI sifts through vast amounts of data to uncover hidden connections between existing drugs and rare diseases, significantly speeding up the journey to

Have you ever felt that living with a rare or chronic illness comes with an unseen weight, a battle fought not just in the body, but in the mind too? 🧠In this episode of March Forward, we gently explore the profound emotional and psychological impacts that often accompany rare and chronic conditions. We discuss the often-arduous "diagnostic odyssey" and how it, along with the daily realities of illness, can lead to feelings of sadness, anxiety, and a deep sense of grief for the life that was

The search for a rare disease diagnosis can be a long and lonely journey. But what if advanced technology could help light the way?In this episode of March Forward, we’re exploring SHEPHERD, an innovative AI approach bringing new hope to rare disease diagnosis. Published in June 2025, SHEPHERD uses a clever "few-shot learning" method, allowing it to learn effectively even from limited patient information. It also uses a deep rare disease knowledge graph, acting like a vast, interconnected map to

Is inflammation the hidden key in your rare disease journey? 🤔For millions living with these complex conditions, understanding the body's fiery response is absolutely crucial to managing progression and finding relief. 🔥In this episode of March Forward, we dive deep into how inflammation impacts rare diseases, exploring its fundamental nature from unseen triggers to its potential for profound, long-term effects on your well-being.We'll unpack the intricate pathways of inflammation, including the

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, the first-ever

What if the the cutting-edge science behind Beqvez, including its AAV vector and the high-activity FIX-Padua variant, relentless cycle of infusions for Hemophilia B could finally pause, allowing your own body to take the reins? 🩸➡️ what clinical trials revealed about its potential to reduce bleeding rates and reliance on prophylaxis ❤️, and the crucial patient eligibility criteria like💪In this episode of March Forward, we explore Fidanacogene Elaparvovec (brand name: Be neutralizing antibody

What happens when standard treatments for a rare cancer like synovial sarcoma fall short? Could 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 protein often

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 this