Issue #10 – September 25, 2025

Central Texas Guide to Rare Neuro Updates, Advocacy, and Support

Hey Texas Neuro family,

You asked, we listened. You've told us loud and clear: "Love the info, but make it faster to read." Fair point. Who has time for BIG newsletters when you're juggling doctors' appointments, insurance calls, and everything else life throws at rare disease families?

So here's what's different starting today: same great Texas-focused intel, less words, twice the impact. Think of it as the "highlights reel" version—all the breakthroughs, trial updates, and resources you need, without the fluff.

Here's our promise: Quick to read, maximum value delivered. If something's not helping you navigate this rare disease journey better, it's not making the cut.

Thanks for sticking with us through the format evolution. Now let's get to the good stuff.

Stay fierce.

✅ Texas Medicaid now covers gene therapy - Life-saving treatments accessible regardless of income
✅ New CIDP treatments show 88% success rates - Better outcomes with fewer side effects
✅ 3 major clinical trials enrolling in Texas - Free treatment access for qualifying patients
✅ Houston reaches 10,000 gene therapy milestone - Proven infrastructure for experimental treatments

⏱ Read time: 5 minutes

What happened: Texas Medicaid joined the federal Cell and Gene Therapy Access Model, making million-dollar gene therapies accessible to low-income families for the first time.

Why it matters: Previously, these breakthrough treatments were limited to patients with premium insurance. Now, 84% of Texas Medicaid beneficiaries have potential access to life-saving gene therapies.

What's next: While initially covering sickle cell disease, federal officials indicate expansion to neurological conditions is coming.

🎯 Action for you: Contact your neurologist about future gene therapy options for your condition.

The breakthrough: Riliprubart, a targeted complement inhibitor, preserves immune function while treating CIDP - something traditional therapies can't do.

By the numbers:

Texas advantage: Multiple medical centers now participating in Phase 3 trials, offering free access to participants.

📍 Find Texas trial sites:

CAPTIVATE Study - No placebo period, all participants receive active treatment

ARISE Study - Open to newly diagnosed and experienced patients

🔗 Quick enrollment check: Call GBS/CIDP Foundation clinical trial hotline.

Texas Children's Center for Cell and Gene Therapy treated its 10,000th patient - representing one of the world's largest experimental treatment programs.

What this means for families: ✓ Established safety protocols for complex treatments
✓ Insurance navigation support
✓ Family support services during treatment
✓ Direct access to cutting-edge therapies

🎯 Access point: Texas Children's Clinical Trials Office - 832-824-4000

Scottish Rite for Children received $420,000 from NIH to develop the first FDA-approved treatment for Childhood-Onset Striatonigral Degeneration (SNDC).

The impact: SNDC affects 1 in 1,000,000 children, with most losing ability to speak or move by age 5. This Texas-led research offers the first hope for treatment.

Patient story connection: Families like the UT Health San Antonio team who started Channeling Hope Foundation show how determined parents accelerate rare disease research.

"Thanks to your trial spotlight, I enrolled in the CAPTIVATE study and am seeing improvements for the first time in years." - Maria S., Houston

"The Medicaid gene therapy news gives our family hope we never had before." - Robert T., Austin

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October 15-17: NALCN International Conference, San Antonio
November 8: MDA Conference Results Release
December 1: Holiday Treatment Planning Webinar

🔔 Stay informed: Subscribe to our weekly updates at texasneurorare.org

📧 Stay Connected to us at [email protected] our trusted admin email.

MEDICAL DISCLAIMER: This newsletter provides educational information reviewed by our medical advisory board. Content is not intended as medical advice. Always consult your healthcare provider before making treatment decisions. Clinical trial information is provided for educational purposes - eligibility and enrollment should be discussed with qualified medical professionals.

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