The Shadow
Searching for Cures
February 28 is recognized as Rare Disease Day.
Accordingly, numerous advocacy groups gather in DC to lobby lawmakers about rare disease research, treatments and potential cures.
Such rare diseases that generate advocacy interest include Lou Gehrig’s Disease (also known as ALS), Huntington’s Disease and other neurodegenerative diseases.
Individuals who are affected by these rare diseases often have no places to turn to treat their debilitating illness.
Given this reality, family members and loves ones are left feeling frustrated, saddened and hopeless.
Even before I became Health Subcommittee Chairman, I heard these heartbreaking stories.
A swimming friend of mine in the Roanoke Valley, Rob Lawson, died from his battle with ALS in 2022.
One of my mentors, former Virginia House of Delegates Republican Raymond R. Robrecht (Ray) of Salem died from ALS on Christmas Eve in 1994.
I grew up with one family that had Huntington’s Disease, and subsequently I have learned of a local lawyer who is valiantly battling it as well.
Huntington’s Disease is a genetic disorder where half of the family is expected to carry the debilitating gene.
Unfortunately, these diseases claim the lives of colleagues, family members and friends.
On February 19, Americans received the sad news that actor Eric Dane died from his battle with ALS.
Dane was best known for his role as Dr. Mark Sloan (also known as McSteamy) in Grey’s Anatomy.
In April 2025, Dane announced his ALS diagnosis. He had struggled with symptoms for months prior to his diagnosis.
When Dane came to Capitol Hill in September, he brought additional attention to rare disease research.
Accordingly, I met with him to hear his story and how Congress can help. For his visit, Dane was confined to a wheelchair.
I appreciated meeting with him to discuss the importance of advancing policies that support patients who struggle with neurodegenerative diseases.
Dane knew additional research would not save him, but he hoped it could save others.
In the 117th Congress, I co-sponsored the Accelerating Access to Critical Therapies for ALS Act. This bill authorized additional federal funds for groups to research and develop drugs to help treat ALS. It passed, but more needs to be done.
On Huntington’s, in a 2024 Health Subcommittee legislative hearing, I discussed the possibility of using Adeno-associated Virus (AAV) gene-based therapies for inherited diseases.
AAV therapies use engineered, non-pathogenic viruses that are not known to result in human diseases. These viruses deliver genetic material directly into patient cells to treat genetic diseases.
Dr. Flotte of UMass Chan Medical School testified at the hearing. He confirmed to me that such gene-based therapies are potentially helpful for the purposes of RNA interference, but it would be necessary that these therapies silence the gene carrying Huntington’s.
As Health Subcommittee Chairman, I am working to advance legislation that tackles Huntington’s Disease, ALS and other rare diseases.
On the same day that I met with Dane, I participated in an Energy and Commerce markup hearing. Of the bills we considered and reported, I voted to advance H.R. 1262, the Mikaela Naylon Give Kids a Chance Act.
This bill reauthorizes the Food and Drug Administration (FDA) Rare Pediatric Disease Priority Review Voucher Program.
This program helps incentivize the development of treatments for rare pediatric diseases.
Additionally, the FDA is empowered to require pediatric cancer trials for new combinations of drug therapies.
After Committee action, the Give Kids a Chance Act was included as a part of the health extenders signed into law on February 3rd.
In early February, I was proud to support the bill and deliver the Give Kids a Chance Act to President Trump!
Later in February, the FDA issued new draft guidance to facilitate the development of individualized therapies for ultra-rare diseases!
This framework has the potential to help bring first of their kind treatments to patients with rare diseases like ALS and Huntington’s Disease.
I am hopeful that this renewed push at the federal level will help in our quest to find successful treatments.
As we acknowledge Rare Disease Day, let us remember those who courageously fought against rare diseases, including Dane, Rob, Ray and countless others.
These individuals will continue to inspire policymakers, agencies and researchers as we continue the quest to find cures for rare diseases.
If you have questions, concerns, or comments, feel free to contact my office. You can call my Abingdon office at 276-525-1405 or my Christiansburg office at 540-381-5671. To reach my office via email, please visit my website at https://morgangriffith.house.gov/. Also on my website is the latest material from my office, including information on votes recently taken on the floor of the House of Representatives.
