Rare blood diseases in U.S
Most mornings, my commute is a 12-minute drive from my home in the hills above Danbury, Connecticut, to the nearby NORD offices. But Tuesday’s commute started early – 5:30 a.m. – with a train ride into New York City and a brisk walk up 42nd Street to the NASDAQ Marketplace in Times Square.
At the Marketplace, my NORD colleagues and I joined other members of the “MPN Coalition” who had gathered to participate in the bell ringing ceremony for the day’s opening of the NASDAQ stock exchange.
We were there because September is Blood Cancer Awareness Month, and MPNs or myeloproliferative neoplasms are rare blood cancers. Three rare diseases classified as MPNs are essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF).
Because these are chronic conditions, the Coalition promotes education and awareness to help patients achieve their fullest potential and enjoy the highest possible quality of life. Many people live full, active lives for many years with these conditions.
Various activities are planned for Blood Cancer Awareness Month, including a conference in Atlanta on Sept. 12, but Tuesday’s bell-ringing was an opportunity for us, as patient advocates, to step outside of our usual roles and view the rare space from another perspective – that of the companies and investors involved in orphan product development.
It’s good to be reminded that developing drugs and biologics is a high-risk business, and developing orphan drugs and biologics poses particular challenges. The rare disease community can work together more effectively when we all have an opportunity from time to time to walk a few steps in each other’s shoes.
The bell-ringing was organized by Incyte Pharmaceuticals, which developed an orphan product that was approved a few years ago for one of the MPNs. Incyte invited the Coalition members – the MPN Education Foundation, MPN Research Foundation, MPN Advocacy and Awareness, Leukemia & Lymphoma Society, CancerCare, Cancer Support Communities and NORD – to participate.
So, instead of my usual morning routine, which would include phone calls and emails from patients and caregivers desperately seeking a treatment or cure, I found myself cheering and clapping for the launch of another busy day in the nation’s financial markets. It was a good reminder that those patient calls for help are being heard in places and ways that aren’t always obvious to us.
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