NORD Press Release on approval of Ensuring Access to Clinical Trials

The Kennedy's Disease Association (KDA), as well as hundreds of other organizations, supported the passage of S. 139. Many of us living with Kennedy's Disease, as well as other Rare Disorders, also contacted their Senators and Representatives asking for their support.

NORD Issues Statement Applauding the Approval of Ensuring Access to Clinical Trials Act

Washington, D.C.—September 29, 2015—The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), on yesterday’s approval of the Ensuring Access to Clinical Trials Act (S. 139) in the United States House of Representatives.

The House of Representatives echoed the U.S. Senate and showed its commitment to 1 in 10 Americans and their families by passing the Ensuring Access to Clinical Trials Act (S.139).  By passing S. 139, the House voted to remove income-related barriers to participation in clinical trials and toward developing much-needed treatments for the 7,000 known rare diseases, only a few hundred of which currently have FDA-approved treatments.

Today, 30 million people and their families have added hope that their tremendous unmet medical needs are one step closer to being addressed. Studying one rare disease can often lead to understanding of other rare diseases, as well as understanding of more common diseases.

As the Ensuring Access to Clinical Trials Act moves to the President’s desk, we close in on eliminating the challenges of rare diseases and to stopping them from altering and ending the lives of too many Americans much too soon.  NORD is proud to have supported this bill and to have advocated for its passage with the Cystic Fibrosis Foundation, Muscular Dystrophy Association, and many others in the rare disease community.

Peter L. Saltonstall
President and CEO, National Organization for Rare Disorders (NORD)

Read more about the Ensuring Access to Clinical Trials Act here.

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About the National Organization for Rare Disorders (NORD)®
Established in 1983, the National Organization for Rare Disorders (NORD)® is the primary nonprofit organization representing all patients and families affected by rare diseases in the U.S.  NORD is committed to the identification, treatment and cure of all 7,000 rare diseases that affect 30 million Americans, or one in every 10 people.  NORD provides programs of advocacy, education, research and patient and family services to improve the lives of all people living with rare diseases. NORD represents more than 230 disease-specific member organizations and collaborates with many other organizations in specific causes of importance to the rare disease patient community. Join NORD at www.rarediseases.org and on Twitter at @RareDiseases.

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The MDA also made an announcement today regarding the passage of this act. You can read the announcement at this link:  http://cqrcengage.com/mda/advocacy/issues/eact

Greatest Regret

I read an interesting article by Rachel Gillett this morning. It was a synopsis of a study of 1,500   

people 65 and older who were asked what their greatest regret in life is.

Link to article:  http://finance.yahoo.com/news/americans-over-65-shared-greatest-120000285.html

“What do you regret when you look back on your life? That's what Karl Pillemer, professor of human development at Cornell University, founder and director of the Cornell Institute for Translational Research on Aging asked his study subjects. … he refers to his subjects as ‘the experts’ because they hold more trued-and-true wisdom than any self-help book or pundit could possibly offer. 

… Pillmer writes on Quora that he expected ‘big-ticket items’ like affairs, bad business deals, or addiction as his experts’ biggest regrets.  

… He was therefore unprepared for the answer they often gave:

I wish I hadn't spent so much of my life worrying

… A critically important strategy for regret reduction, according to the elders we interviewed, is increasing the time spent on concrete problem solving and drastically eliminating time spent worrying. One activity enhances life, whereas down the road the other is deeply regretted as a waste of our all-too-short time on Earth.”

Many of us living with Kennedy’s Disease or other conditions that currently do not have a treatment or cure, have probably gone through and occasionally still go through moments of concern – wondering what tomorrow will bring. It is natural to fear the unknown. Yet, as many of these 1,500 people commented, worrying didn’t do any good.

Perhaps Bobby McFerrin knew what he was singing about with his 1988 song, "Don't Worry - Be Happy." I'm humming the song right now as I post this. It sure is a catchy tune.

NSAID – possible new risks

A recent article in the Costco Connection was interesting. It explained that the Food and Drug Administration (FDA) has issued new warnings on the use of nonsteroidal anti-inflammatory drugs (NSAIDs). The new language used by the FDA is for prescription products, but they are also looking into over-the-counter products as well. Currently over-the-counter products warn about the risk of heart attack or stroke with increase dose and duration of use.

The new warnings for these products are:

• The risk of heart attack or stroke can occur as early as the first weeks of using NSAIDs. The risk may increase with longer use of the product.
• NSAIDs can increase the risk of heart attack or stroke in patients with or without heart disease or risk factors associated with heart disease.

The article summarized the warnings in this way. “In order to balance the benefits versus the risks, the short answer offered by most medical professionals is: Take as small a dose of medicine as is appropriate and/or prescribed, and take it for only as long as you need it or are directed to take it.”

As always, talk to your health care professional or pharmacist if you have questions or concerns.