A rare chance to help those in need

Recently the CIRM Board voted to support the creation of a Rare Disease Advisory Council (RDAC) in California. An RDAC is an advisory body providing a platform for the rare community to have a stronger voice in state government. They address the needs of rare patients and families by giving stakeholders an opportunity to make recommendations to state leaders on critical issues including the need for increased awareness, diagnostic tools and access to affordable treatments and cures.  

California is now in the process of creating an RDAC but, as a recent article in STAT highlighted, we are far from the only one.

Guadalupe Hayes-Mota

21 states give rare disease patients a seat at the table. The other 29 need to follow suit
By Guadalupe Hayes-Mota Originally published by STAT on July 26, 2021

A powerful movement is taking shape in the U.S. rare disease community that could transform the lives of millions of people. That’s right — millions. Even though a single rare disease may affect only a few individuals, there are several thousand of these problematic diseases that are difficult to identify and treat.

Since 2015, 21 U.S. states have passed legislation to create Rare Disease Advisory Councils that provide platforms for patients and family members to communicate with experts, policymakers, and the broader public. It’s critical to seize this hopeful moment because the needs of so many people living with rare diseases go unaddressed.

I know because I’m one of them.

I was born and raised in a small town in Mexico and diagnosed at birth with hemophilia, a rare genetic disease that prevents the blood from clotting after trauma or injury. While treatment existed in other parts of the world, I had only limited access to it, forcing me to live an isolated childhood indoors, protected and isolated from the world.

When my appendix burst at age 12, I underwent emergency surgery, followed by a desperate eight-hour ambulance ride to a hospital in another town in search of better medication to stop the bleeding. Doctors told my parents I was unlikely to survive, but against all odds I did — after clinically dying twice in the operating room. I am one of the few lucky people with my condition to have survived severe bleeding events without treatment.

After this traumatic incident, my family moved to a small town in California’s Mojave Desert. Navigating the health care system as an immigrant and not knowing the language was complicated. Accessing treatment and services for my disease was almost impossible at first. The nearest specialist was 90 minutes away. Thankfully, with help from the hemophilia association chapter in our area, I gained access to care and treatment.

Read the complete article here.

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