📝 Editor's Note: The Floodgates Are Opening

Happy Thanksgiving, Texas Neuro family,

For thirty years, CIDP patients had mainly two treatment options: steroids and IVIg. That's was pretty much it. Some found these didn’t work or were just not enough. I was in the stampede for more options myself. Why not? Read on and see more about our hope and get in on your own stampede!

In just the past 18 months, three new drug classes entered trials or won approval. CAR-T trials are opening for stiff person syndrome. MG just got its fourth targeted therapy in two years.

The science has finally caught up. This issue covers some of what matters most—because it's happening in our conditions.

📋 QUICK READ

CIDP: First new drug class in 30+ years (efgartigimod approved)
MG: Fourth targeted therapy approved (nipocalimab)
CAR-T: Trials opening for SPS, MG, CIDP
LEMS: Now in national cancer screening guidelines

Read time: 4 minutes

COMING SOON: A NEW RESOURCE

Neuro AI Ally: Helping Central Texas neurological care teams make sense of AI - without the hype or the headaches. Introducing the handshake between us - as humans - with AI - our future. We translate what's actually working (and what’s not), what's coming next, and how to implement it without losing the human touch that makes healthcare matter. Practical guidance for busy people - from the Patient to the Professional, delivered in plain English.

💊 CIDP: First New Drug Class in 30 Years

The FDA approved efgartigimod (Vyvgart Hytrulo)—the first FcRn blocker for CIDP and the first new drug class since IVIg became standard.

Why it matters: Instead of hours in infusion chairs, it's a 30-90 second weekly injection. The ADHERE trial showed 61% lower relapse risk versus placebo.

More coming: Batoclimab showed 84% responder rate in Phase 2b. Riliprubart is in Phase 3 across 28 countries. Experts predict "significant expansion of options within 3-5 years."

🎯 Ask your neurologist: "Am I a candidate for efgartigimod?"

💉 MG: Another Targeted Option

FDA approved nipocalimab (Imaavy) for generalized MG in patients 12+—the first FcRn-blocking monoclonal antibody for the condition.

The bigger picture: Since 2017, MG has gained complement inhibitors (eculizumab, ravulizumab, zilucoplan) and FcRn inhibitors (efgartigimod, rozanolixizumab, now nipocalimab). A decade ago? None of these existed.

Breaking: UNC research confirms efgartigimod works for ALL MG subtypes—not just AChR-positive. This opens treatment to MuSK-positive and seronegative patients.

🎯 Ask your neurologist: About FcRn inhibitors and complement inhibitors by name—don't assume they'll volunteer all options.

🧬 CAR-T: The Immune Reset Arrives

CAR-T therapy—engineering your own immune cells to fight disease—is showing remarkable results in autoimmune conditions. In one trial, 94% of patients remained off immunosuppressive therapy.

What's being tested:

  • Stiff Person Syndrome: Kyverna expects Phase 2 results early 2026, with potential FDA filing that year—possibly the first CAR-T approved for ANY autoimmune disease.

  • MG & CIDP: Multiple trials underway. Bispecific CAR-T cells showed improved function in treatment-resistant CIDP.

Why this matters: Unlike ongoing infusions, CAR-T is one-time. The goal isn't symptom management—it's eliminating the immune dysfunction causing your disease.

🎯 If you have refractory disease: Ask about CAR-T trial eligibility at major academic centers.

⚡ QUICK HITS

LEMS in Cancer Guidelines: NCCN now recommends VGCC antibody testing for lung cancer patients with proximal weakness. Studies suggest 90% of LEMS cases in cancer patients go undiagnosed.

MOGAD Treatment Gap: New study shows tocilizumab outperforms rituximab in MOGAD—important for patients who haven't responded to standard treatment.

GBS Pipeline: ANX005 (complement inhibitor) in Phase 3. Case reports show efgartigimod working in acute GBS. First new options in 30 years may be coming.

Autoimmune Encephalitis: Ofatumumab showing promise in refractory cases. Third-line options (bortezomib, tocilizumab) emerging for patients who fail standard treatment.

🌵 TEXAS RESOURCES

UT Physicians Neuromuscular (Houston): (832) 325-7080
Houston Methodist Trials: houstonmethodist.org/neuro
GBS/CIDP Foundation: 1-866-224-3301
MG Foundation: 1-800-541-5454

🎯 THIS WEEK'S ACTIONS

  1. CIDP/MG patients: Ask about newer targeted therapies by name

  2. Refractory disease: Ask about CAR-T trial eligibility

  3. Unexplained weakness + dry mouth: Request VGCC antibody testing

  4. Everyone: Share this with your neurologist—they may not know all options

📖 TERMS TO KNOW

FcRn Blocker: Reduces harmful antibodies without suppressing entire immune system (efgartigimod, nipocalimab)

Complement Inhibitor: Blocks immune pathway causing nerve damage (eculizumab, zilucoplan)

CAR-T: One-time treatment using your modified immune cells to "reset" autoimmune dysfunction

Refractory: Disease not responding to standard treatments—may qualify for newer options

💡 BOTTOM LINE

CIDP has its first new drug class in 30 years. MG has four targeted therapies. CAR-T trials are opening for conditions we thought would never have precision treatments.

The science caught up. Make sure your care does too.

FROM OUR NETWORK

RarelySerious Newsletter www.rarelyserious.com

Because sometimes you need to laugh about the absurdity of rare disease life—the 47-minute hold times, the "have you tried yoga?" suggestions, the insurance denials for medications that cost more than your car.

Dark humor. Zero medical advice. All the things you're already thinking.

⚠️ Disclaimer

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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