My name is Mousumi Bose, and I am the mother of Ilan Betzer. Ilan was born in August 2010 and was soon diagnosed with Zellweger spectrum disorder, a rare peroxisomal disorder that is categorized as a leukodystrophy. As his disease progressed, our journey with Ilan was marked by complex challenges. Ilan passed away just after his first birthday in October 2011. He touched so many lives during his short life, and still continues to do so to this day.
As there are few treatment options for peroxisomal disorders, care was primarily symptom-based. And there were a lot of symptoms to manage. Furthermore, because there is so little known about peroxisomal disorders compared to more common diseases, many medical care providers would make assumptions about the disease course, and often prescribe care that was not beneficial and sometimes risky. It was these alarming experiences that really shaped my desire to contribute to this limited knowledge base about rare diseases through my own research.
I learned about the ULF when I started getting involved in patient advocacy. I was involved with a newly formed advocacy organization for peroxisomal disorders, and the leadership of this organization looked to the ULF as a longstanding model for patient advocacy in the rare disease community.
I think one of the biggest impacts that the ULF has had on me, personally, was when our organization collaborated with the ULF for the 2015 International Peroxisome and Leukodystrophy Meeting. Leveraging the wide reach and sustainability that the ULF has maintained, our organization was able to gather some of the most notable researchers in the field to collaborate on new research supporting more effective treatments. The magnitude of this meeting, which was made possible by the ULF, was so important in helping me feel hopeful about the future care for peroxisomal disorders and leukodystrophies.
Today, you have an opportunity to help the ULF continue its work. Your contributions allow ULF to support critical research, connect families with life-changing resources, and raise awareness for rare diseases like leukodystrophy and peroxisomal disorders. Your generosity strengthens our community and brings hope to families like mine.