Hope is often the driving force behind the relentless work of research teams dedicated to developing transformative therapies for the millions of individuals impacted by rare diseases—95% of whom still lack effective treatment options. The introduction of specialty pharmaceuticals and cell and gene therapies has brought newfound optimism, with the potential to slow disease progression or even cure these conditions. However, the high costs, limited distribution, and complex reimbursement structures surrounding these therapies limit availability and access.
