fbpx

Category: Chris Duane

To Representative Ferguson

Posted on by caseyscurefoundation

To Representative Ferguson

From: Christine G. Duane, Executive Director, The Foundation for Casey’s Cure, Inc.
RE: The Rare Disease Community of Georgia
Date: March 2, 2021

Dear Representative Ferguson,
On Tuesday, March 1, I had the opportunity to speak with your staff member, Robert Redding.
The purpose of our conversation was to provide him with information about why you, Rep.
Ferguson should support federal legislation that will improve access to care, services, and research that will improve the lives of the rare disease community of Georgia, especially within
your district. This is the text of my presented speech to him. On behalf of the Rare Disease Community across Georgia and the rest of the United States, 25 million patients are calling for your support. I hope you heed their call on their behalf.

Sincerely,
Christine Duane, Executive Director of The Foundation for Caseys Cure, Inc.
162 Wellington Drive
LaGrange GA 30241
732-300-5708

The text of our call was as follows:

Hello!
My name is Chris Duane from LaGrange/Troup County, Georgia.
In preparation for this meeting, I’ve spent some time reviewing the health-related bills and resolutions previously or currently supported by Representative Ferguson. In doing so, I’d like to commend Representative Ferguson for his support for:
• HR 119: Lower Cost More Cure Act of 2021
• HR 4066: Save The Rural Hospitals Act of 2021
• HR 3630: The Lymphedema Treatment Act and
• HR 1916: Ensuring Lasting Smiles Act

Representative Ferguson’s support for these and other health issues leads me to my first ‘ask’ for today’s meeting.

As an active member of the Rare Disease community, I hosted an event at Lafayette Square on Monday, February 28, in support of #RareDiseaseDay. I was honored to hear the tragic stories of my neighbors in LaGrange and those who live in your district and their struggles with getting help for their very sick child or themselves. It is on behalf of these people that I urge Representative Ferguson to demonstrate his commitment to the Rare Disease community and join with 100 fellow House members of the nonpartisan bicameral Rare Disease Congressional Caucus. The Rare Disease community’s voice needs to be heard and we are looking for his voice to join with ours.

One of the other resolutions he supported was HR 3537, Accelerating Access to Critical
Therapies for ALS Act, which was introduced to the House floor on May 25, 2021, and moved for Presidential signature on December 23, 2021, to become PL 117-79. This bill was passed with unprecedented speed; a credit to the writers and supporters of the ALS community. This leads me to my second ask: The Rare Disease community strongly urges Representative Ferguson to support the Speeding Therapy Access Today Act of 2021, more commonly known as theSTAT Act (HR 1730). This bill is a bipartisan bill, created with the input of the Rare Disease community, aimed at improving the development of and access to therapies for the Rare Disease community.

Specifically, the STAT Act of 2021 (HR 1730) will enact policy reforms at the FDA level to accelerate the development of therapies across the rare disease spectrum. And, just as the Critical Therapies Act did for ALS, the STAT Act of 2021 has the huge potential of shortening the development process of a rare disease patient from an average of 15 years to as few as 3-4 years.

25 million Americans deserve access to therapies within their lifetime. Many of these
Americans will die before those therapies are made available to them or their family members.

Five years ago, the FDA established the first FDA Center of Excellence focused on
oncology, which has been extremely successful in bringing new cancer therapies to
patients. We ask for the same opportunity to be given to the Rare Disease community.
We ask you to show your support for the Rare Disease community in your district and
cosponsor the STAT Act of 2021, HR 1730.

Thank you and respectfully ask for your support on these legislative issues.
_______________________________

Dear Reader,
If you would like to receive updates on the legislative progress we are achieving with our representatives from Georgia, please sign up for our newsletter. We promise not to fill your inbox with junk…??‍♀️ ? Read “About Us” to learn more about Casey’s journey with muscular dystrophy.

Legislative Updates

Being The Mom …

Posted on by caseyscurefoundation

By Chris Duane, Casey’s mom…

So often I want to write about how it feels to take on the project of finding a treatment and ultimately a cure for my daughter’s rare form of muscular dystrophy. But I also think that you may wonder about our goals; Where are we going? What is your donation money being used for? Why is it important to donate to our cause?

Hopefully, this blog post will be a start at answering some of those questions.

You may well ask, “What makes her disease subtype rare?” In the medical research field, diseases that have less than 2,000 cases (in the United States) are considered ‘ultra-rare’.

You may also ask, how exactly does that help or hinder our ultimate goals? I have come to learn that when the US has an insufficient population of a certain disease, finding funding for it can be super-challenging.

But I’ve also learned this year that the FDA doesn’t really like groups of less than, say, 2,000, to be studied because the impact of the research, even if remarkably successful, won’t help enough people to warrant the money and the work. (I wonder if they look at the cost benefit analysis of getting my daughter healthy as opposed to the outrageous cost it will take to keep her alive for as long as possible? And that the work may well yield information to help another rare disease?)

As you can imagine, belonging to a group that is deemed ‘insignificant’ doesn’t sit well with many people with rare diseases.

So at this point in this post, I would like to tell you about an organization I’m learning from that fosters relationships with legislators who can really make an impact in the rare disease world. It’s called The National Organization for Rare Diseases (pretty creative right?) https://rarediseases.org/rare-diseases/myopathy-myofibrillar/ This link will take you the page that has a LOT of medical information on it. But if you scroll toward the bottom, you’ll see a bit of information about HMERF (Hereditary Myopathy with Early Respiratory Failure), Casey’s subtype.

Also, another group, Every Life Foundation, sponsors Rare Disease Week on Capitol Hill . Casey’s Cure plans to attend these ‘meetings on the Hill’ in February, 2022, in order to put forth our support behind the HEART BILL, HR 1184. ( We attended virtually in 2021.)

This bill has a section in that could make a HUGE difference in the research opportunities for ultra-rare diseases like Casey’s.  The text of this bill that is relevant to Casey’s Cure is the section that addresses examining and including research from the European Union. This is important because ultimately it will allow aggregation of small pockets of research that, when aggregated, would create a group large enough that would qualify for FDA studies/research/grants, etc. ( https://www.congress.gov/bill/117th-congress/house-bill/1184/text#H1C190F0C28FE4AC1AAD4AC6C37D71649 )

I intend to do a better job of keeping those interested abreast of our work throughout the coming months. Our hill is a steep one, but as long as there is hope, there is work to be done.

So very gratefully yours,
Chris