WASHINGTON D.C. (February 24, 2026) This year, we arrive in Washington D.C. to advocate for our vision for rare disease families: a world where all rare disease families can access multiple safe and affordable treatments.
We are visiting DC for the first time since the accelerated approval of FORZINITY, the first treatment approved for individuals with Barth syndrome weighing over 30 kg (~66 lb). That approval was a milestone, but we aren’t done yet. We want every family who can clinically benefit from FORZINITY to have safe, affordable access. Currently, that’s not guaranteed.
Importantly, our hopes go beyond FORZINITY.
We are working toward a world where regulatory pathways are predictable and appropriate for ultra-rare diseases, where families can choose from a range of safe and effective therapies, and where Barth syndrome and other rare diseases don’t limit the lives of our community members.
What Is Rare Disease Week?
Rare Disease Week exists because the rare disease community built it. Families don't come to Washington to ask for a seat at the table. They come because they made the table.
No one understands what is needed more than the people living it. Families know when a policy is working and when it isn't. They know when insurance coverage or denial will change their life. They know the difference between a drug being approved and a drug being accessible.
During Rare Disease Week, families take that knowledge directly to the people with the power to act on it. The Barth Syndrome Foundation and our community members will be in Washington with specific asks, stories, and a clear message: science and advocacy can build a world that’s a little bit easier for rare disease families.
What We Hope For From Our Leaders
Here are a few of our answers to the leaders who ask "what can we do?" (and we’re so grateful when they ask!):
- Expand access for all who need it. Right now, children under 30 kg cannot access FORZINITY through standard channels. This means many of our most medically fragile children may not be able to access treatment. We need full label access now, and we need a regulatory approach that does not segment tiny communities.
- Make accelerated approval truly accelerated for all rare disease communities. Accelerated approval processes must be efficient and effective. It took us over six years to get accelerated approval for FORZINITY. We hope for regulatory pathways that deliver data-driven, live-saving treatments to families quickly.
- Provide legislative support for post-market and insurance issues. Post-market regulatory requirements place undue burden on our community. Insurance companies have the power to deny coverage and place unreasonable requirements on families. We need congressional support to make sure approval translates to access.
What We Hope for From the Future
We come to Rare Disease Week to hold our leaders to the same standard we hold ourselves: do what the evidence supports, act without unnecessary delay, and center the needs of families. Barth syndrome is rare. The need for action is not.
You can read more about Rare Disease Week here.
