Progress in research and treatments is a reason for holding on to hope
And hope is what keeps the PNH community connected
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If there’s one thing living with paroxysmal nocturnal hemoglobinuria (PNH) teaches you, it’s that life rarely follows the script you wrote for it. For many of us, PNH didn’t just change our health; it changed how we measure time, how we define strength, and how we celebrate the quiet victories that most people never even notice.
This year, conversations around PNH have continued to center on three powerful themes: treatment progress, quality of life, and the future of cure-focused research. And while medical science keeps pushing forward, the emotional and human side of living with PNH is just as important.
For many of us, today’s treatments have transformed what living with PNH looks like. Where fear of constant hemolysis, transfusions, or life-threatening clots once dominated our daily lives, many are now seeing longer stretches of stability. That matters. It means being able to show up for work, attend family events, and plan months ahead, instead of hours. It means reclaiming pieces of normalcy that once felt out of reach.
Moving forward
But stability doesn’t mean PNH disappears. The fatigue can still linger. The mental weight of managing a rare disease can still feel isolating. And there’s always that quiet awareness in the background that your body is doing something most people will never have to think about.
This year has also brought continued excitement around next-generation therapies. Longer-acting treatments, oral options, and new complement pathway targets are expanding what care might look like in the future. This is not only a win for convenience, but also for our freedom. It would mean fewer infusions, less disruption, and more life lived outside of treatment schedules.
Then there’s the question many of us carry in our hearts after a diagnosis: Will there ever be a cure outside of a bone marrow transplant?
A transplant remains the only known cure today, but it carries significant risk. Research into gene therapies and immune system modulation is growing, and while we’re not at the finish line yet, the direction research is going feels more hopeful than it has in decades. And hope matters. Hope is what helps you show up for your family when you’re tired. Hope is what helps you advocate for yourself in doctors’ offices. Hope is what keeps the PNH community connected — sharing stories, research updates, and encouragement across miles and time zones.
Living with PNH teaches resilience in a very quiet, personal way. It teaches patience. It teaches gratitude for “boring” lab results. It teaches you that strength isn’t always loud; sometimes it’s just choosing to keep moving forward.
Note: PNH News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of PNH News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to paroxysmal nocturnal hemoglobinuria.
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