Treatment for Rare Blood Disorder Provides Hope for the Future
Dipak from Nepal has always been passionate about music. Ever since he was young, he dreamed of creating songs, performing, and making people happy through his art.
“It’s not just a hobby, it is who I am,” he said.
Then, he was diagnosed with two rare and debilitating blood disorders – aplastic anemia, where bone marrow stops producing enough red blood cells, and paroxysmal nocturnal hemoglobinuria (PNH), which causes red blood cells to break apart.
Dipak’s life became a cycle of hospital visits, tests, and treatments. He needed blood transfusions once a month, and sometimes every 15 days.
“My life revolved around my illness,” he said. “I was constantly exhausted, physically weak, and emotionally drained. Instead of building my music career, I spent my days wondering when I would need my next transfusion.”
One day, his doctor told him about a new medication that could help. At the end of 2024, The Max Foundation expanded its partnership with Novartis to include access to treatment for PNH. Now, patients in Nepal are getting the care they need and physicians are receiving training that provides a framework to better understand the disease.
However, having both aplastic anemia and PNH made treatment more complex. Dipak’s case required more testing and review by the International PNH Interest Group, a professional society Max has partnered with since 2024. They’re dedicated to enhancing knowledge about PNH and improving outcomes for patients in low-resource countries.
After a thorough evaluation, Dipak was able to start treatment. Now, four months later, he has more energy and freedom than he’s had in years.
“Most importantly, I am finally able to focus on music again,” he said. “I spend my time practicing, performing, and working toward the career I had once believed was slipping away. My illness no longer defines every decision I make.”
Dipak said he’s also grateful for the support. His medical team was with him every step of the way, and they encouraged him not to lose hope.
“Today, every song I play reminds me of how far I have come. It reminds me of every doctor, every healthcare worker, every member of the Max team in Nepal, and every person who believed in me when I struggled to believe in myself,” Dipak said. “Medication didn’t just change my treatment, it changed my life. It gave me the strength to pursue my dream again, and the opportunity to live the life I had always hoped for.”
