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.

You might also like
BLOOD PRESSURE DISEASES | Blood Pressure Diseases and the
BLOOD PRESSURE DISEASES | Blood Pressure Diseases and the ...
Rare, serious respiratory virus sickening kids across Midwest
Rare, serious respiratory virus sickening kids across Midwest
Rare Blood Moon Rises During Upcoming Total Eclispe 15,2014
Rare Blood Moon Rises During Upcoming Total Eclispe 15,2014

Dragsbaek: 5 public health threats scarier than Ebola  — Longview News-Journal
Children died not from a rare infectious disease but from a vaccine-preventable disease that we didn't protect them from. 2. Pertussis: In 2013, Texas reported 3,985 cases of pertussis, or whooping cough — more than any other state in the U.S.

Prenatal Multivitamin - Vitamins for Pregnancy - Vitamins for Pregnant Women - For Women Who Want the Best Nutrition for Their Babies to Come - The Good Health of Your Baby Starts with Good Nutrition Before Birth - Have Little Time to Buy Enough Balanced Meal for You and Your Baby? Supplement Your Nutrition with Multi-vitamins
Health and Beauty (Premivita)
  • A Multiple Vitamin/Mineral Supplement for Pregnant and Nursing Mothers with Enzymes, Amino Acids and Herbs.
  • Iron is an important blood building nutrient for the baby as well as for the mother. A deficiency of iron means that new blood cells will be unable to transport...
  • Premivita PrenatalTM supplies 13 valuable minerals that are especially important during the prenatal period. Levels of these minerals in the Premivita PrenatalTM
  • Premivita Prenatal Vitamins had even B12 as Methylcobalamin, which is so rare to find in prenatal vitamins on the market. Anemia specifically caused by vitamin B12...
  • Premivita Prenatal Vitamins had even B12 as Methylcobalamin, which is so rare to find in prenatal vitamins on the market. +Vitamin B12 Deficiency is commonly misdiagnosed...
RARE DISEASE DAY 2013 Rally at the Texas State Capitol
RARE DISEASE DAY 2013 Rally at the Texas State Capitol ...
Rare Disease Day 2013
Rare Disease Day 2013
What is Rare Disease Day?
What is Rare Disease Day?

Popular Q&A

avatar
How can changes in blood pressure cause a stroke?

High blood pressure can lead to heart failure. If the heart isn't pumping properly than the chance of forming a clot increases and if this clot breaks free and makes it to the head than a stroke will occur

Related Posts