💥 100% response rate in myasthenia gravis - Every patient got better. Every. Single. One.
🧬 Gene therapy delivered directly into a child's brain - She's developing normally now
⚡ FDA just cut years off approval timelines - Ultra-rare treatments moving at lightning speed
🏥 Texas became the epicenter - Our medical centers are making national headlines

⏱ Read time: 4 minutes | Then forward this to every rare disease family you know

Twenty years ago, if you got diagnosed with CIDP, myasthenia gravis, or GBS, your neurologist handed you a pamphlet and said "we'll manage symptoms as best we can."

Five years ago, they added "there are some trials, but they're experimental."

This year? They're saying "we might be able to cure this." It may not be my disease or yours but as the dominoes fall we all get a little close.

That's not incremental progress. That's a revolution. That’s kick the barn door down stuff!

And it happened in Texas while you were fighting insurance companies, explaining your condition to your another new specialist, and wondering if anyone was actually working on this stuff.

They were. And the results just dropped.

Read that number again: 100%.

Not 85%. Not "most patients." Not "statistically significant improvement."

Every single patient got better.

By week 12, all treated patients stopped needing their other immune-suppressing drugs entirely. Patients who'd been tethered to infusions for years—planning their lives around treatment schedules, rationing their energy, living in the gap between doses—reported minimal symptoms within weeks of a single infusion.

One treatment. Immune system reset. Potentially years of relief.

This isn't symptom management anymore. This is your body learning to stop attacking itself.

Here's what that actually means:

You don't schedule your life around infusions anymore. You don't wonder if you'll have enough strength to make it through your kid's birthday party. You don't live in the three-day window after treatment before symptoms creep back.

You get your life back.

Phase 3 trials are launching now. UT Southwestern, Houston Methodist, and Texas Children's are building CAR-T infrastructure. If you have MG, call myasthenia.org and get on the trial notification list today. Not next week. Today.

While everyone was watching CAR-T, Dr. Daniel Curry at Texas Children's Hospital did something that shouldn't have been possible.

He delivered gene therapy directly into a child's brain to treat AADC deficiency—a devastating condition where kids can't make the neurotransmitters their brains need to function. Most don't survive past age five.

The child was three years old. Before treatment, she couldn't develop normally. After? She's hitting milestones.

This proved we can surgically fix genetic brain problems. Not manage them. Not slow progression. Fix them.

Texas Children's has now treated over 10,000 gene therapy patients. That's not a typo. Ten thousand. They're leading the nation because they decided to build the infrastructure instead of waiting for someone else to do it.

Once CAR-T proved immune reset was possible, everything accelerated:

November: FDA approved Itvisma—gene therapy for spinal muscular atrophy at any age. One treatment, lifelong benefit.

September: FDA launched fast-track approval for ultra-rare diseases (under 1,000 patients). What used to take 7-10 years now takes 3-4.

August: ANX005 for Guillain-Barré hit 2.4-fold improvement in disability scores. Single dose, six months of benefit.

September: Texas joined the Cell and Gene Therapy Access Model. If you qualify for gene therapy, cost is no longer the barrier.

This isn't coincidence. This is what happens when one breakthrough proves a concept, and suddenly everyone realizes the old rules don't apply anymore.

While other states were forming committees to study feasibility, Texas medical centers made decisions:

UT Southwestern and Houston Methodist launched shared CIDP diagnostic pathways. Result: Texans diagnosed within 12 months had twice the remission rate compared to those diagnosed after two years.

Texas Children's built gene therapy infrastructure that became the national model.

UT Rio Grande Valley deployed AI screening that cut waitlists by 20% in rural areas.

Home infusion networks expanded 45% across rural counties. Families aren't driving four hours for monthly clinic visits anymore.

Telemedicine grew 32% since 2023. You can see a specialist from Amarillo without leaving home.

This happened because we got tired of waiting. It happened because patients, families, and doctors got tired of "we're working on it" and built the infrastructure themselves.

Maria in Houston got into the CAR-T trial after reading our November issue. First time in years she's had hope that isn't attached to a monthly infusion schedule.

A family in Austin connected with Texas Children's gene therapy program through our resource list. They're scheduled for January.

James in Amarillo didn't know home infusion existed until we wrote about it. His daughter doesn't miss school for clinic visits anymore.

These aren't our wins. These are yours.

You showed up to the appointments when you were exhausted. You advocated when doctors dismissed your symptoms. You joined registries, enrolled in trials, and shared your stories so other families wouldn't feel alone.

Every breakthrough on this list happened because patients refused to accept "there's nothing we can do."

Call myasthenia.org TODAY. Get on CAR-T trial notification lists. Phase 3 is recruiting now.

Ask your neurologist about the diagnostic pathway at UT Southwestern or Houston Methodist. If already diagnosed, ask about home-based SCIG to reduce clinic visits.

Contact Texas Children's gene therapy program (832-824-4000). Infrastructure exists. Access exists. Use it.

Ask your primary care doctor for a tele-neurology referral to a major center. Virtual neurology is covered. Distance isn't an excuse anymore.

Join a patient registry. NORD's IAMRARE led to the first FDA-approved therapy developed from patient data. Your data could unlock tomorrow's treatment.

Mark your calendar:
📅 January 15: CIDP Treatment Update Webinar (Houston Methodist)
📅 February 8-10: Rare Disease Week Texas (Austin)
📅 March 1: New NMOSD patient registry launches

UT Southwestern Neuromuscular: 214-645-8800
Houston Methodist Neuromuscular Center: 713-363-7310
Texas Children's Hospital Neurology: 832-824-4000
Texas Medical Transportation (free rides): 877-633-8747
Clinical Trials: ClinicalTrials.gov (filter: condition + Texas)
GBS-CIDP Foundation: 1-866-224-3301
NORD IAMRARE Registry: rarediseases.org

I manage a rare neurological condition myself. I have caregivers (the best in the world). I plan my life around symptoms too.

So when I say 2025 was different, I mean it. For the first time in twenty years, doctors started using words like "cure" and "reset" instead of "management." The FDA moved faster on rare disease approvals. Texas medical centers made national headlines while other states formed committees.

Some rare diseases stopped being death sentences this year. They became problems we're learning to solve.

That doesn't mean it's solved or everyone has access. The fight isn't over.

But the trajectory changed—because this community refused to accept "nothing can be done."

You made it through another year with a condition most doctors have never heard of. That takes real strength.

2026 is already moving: CAR-T infrastructure expanding, gene therapies in pipelines, FDA fast-tracks cutting years off approvals.

Keep pushing. Keep advocating. Keep showing up for each other.

You're not alone in this.

NeuroAI-Ally (Thursdays): For healthcare workers—AI tools transforming EMG interpretation and neuromuscular diagnosis. www.neuroaially.org

RarelySerious (Fridays): Because if we can't laugh at the absurdity sometimes, what are we doing? This week: "Things My Insurance Company Thinks Are Reasonable." [Subscribe for medically inappropriate humor www.rarelyserious.com

Neighborly Note & Human Disclaimer: We are not doctors—always check with your provider before making health decisions. When in doubt, talk to your doc—then come swap stories with us!

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