2016 Rare Disease Day

On March 2nd, Eveline van Veldhuisen Honig and I travelled to Washington, DC and Albany, New York to represent Narcolepsy Network for Rare Disease Day. Eveline is our Executive Director, and I am a member of the Board of Trustees.

2016 New York State Rare Disease advocates meeting with Senator Hannon (R–Nassau County).

Our goals were slightly different for each state, and we shared the same common agenda as our fellow representatives from other rare diseases: show our respective organizations’ commitment to advocating for people with rare disease, and support legislation that will help those people. We also accepted proclamations of Rare Disease Day for each state.

In Albany, we were able to meet with Senator Kemp Hannon (R-Nassau County). Each organization had a table filled with literature, brochures, wristbands, and information about their respective rare disease. We also sat in the Senate chamber for the reading of the Rare Disease Day proclamation. It was a fantastic event, and everyone who made the trip to Albany did a great job representing their organization / rare disease.

Rare Disease advocates on Capitol Hill.

Eveline travelled to Washington, D.C. for Rare Disease week activities, and participated in the Rare Disease Legislative Advocates conference and lobby day on Capitol Hill.

The conference focused on several bills and laws, especially the OPEN ACT (Orphan Products Extension Now), which contains legislation that holds the potential to double the number of treatments available to rare disease patients.

The conference had many interesting topics and speakers. Topics included discussion of the different agencies, such as the FDA, rare disease legislation, tips and tricks for successful lobbying, advocacy on social media, how to work with industry, and more. The next day on Capitol Hill was both exciting and overwhelming. New York State had many rare disease advocates, so it was hard to get any individual attention, but we all left our brochures. Eveline met with the staff of Senator Schumer, Senator Gillibrand, and Representative Maloney.

Eveline, our Executive Director, in Senator Schumer’s office.
From left: Melissa Patterson, Eveline van Veldhuisen Honig.

What is Narcolepsy?

Narcolepsy is a rare sleep disorder that affects approximately 1 out of every 2,000 people. Out of those people, only about 25% are ever diagnosed. It is poorly understood by both the general public and within the medical community. Narcolepsy is considered a socioeconomic disorder, because it impacts the personal and professional lives of people with narcolepsy.

Narcolepsy is characterized by a range of symptoms, which may vary greatly from person to person. The primary symptoms of narcolepsy include:

  • Excessive daytime sleepiness — imagine pulling an all-nighter, this is what a person with narcolepsy feels like every day.
  • Cataplexy — this is a sudden loss of muscle tone, usually triggered by emotions or stress. Cataplexy’s effects can range from a slight sagging of the jaw, to a full-body loss of muscle tone. About 70% of PWN experience cataplexy.
  • Hypnagogic hallucinations — these are essentially dreams that occur before a person with narcolepsy has fallen asleep.
  • Fragmented sleep — narcoleptics who experience this wake up very briefly, many times each night, often without realizing it.
  • Sleep paralysis — the inability to move upon falling asleep and / or awakening.