Welcome to Issue #6. I hoped to start out with a bright and cheery editor’s note but circumstances seemed to decide otherwise. It has been a somber week and it helped me realize my path is hard but not final.

FDA Decision for BTK Inhibitor in MS
Sanofi’s tolebrutinib faces an FDA verdict September 28. Phase 3 HERCULES data reveal a 31% reduction in disability progression for non-relapsing secondary progressive MS compared to placebo.

First Pediatric AADC Gene Therapy in Texas
At Texas Children’s, a 3-year-old received FDA-approved AAV-based therapy for aromatic l-amino acid decarboxylase deficiency. This landmark surgery offers hope to the 350 globally affected children.

Stem Cell Relief for Peripheral Neuropathy
Mesenchymal stem cell infusions produced a 35% improvement in quality-of-life scores for diabetic neuropathy trials, with patients reporting sustained pain reduction and nerve-function gains.

Epilepsy Gene Therapy Milestone
Neurona’s NRTX-1001 earned FDA RMAT designation for drug-resistant epilepsy. Concurrently, fenfluramine shows sustained seizure reductions in Dravet and Lennox-Gastaut syndromes.

Home IVIg Safety Advances
New pump systems with auto-stop and temperature monitoring cut adverse events by 40%, translating to fewer ER visits for home-infusion patients.

Long-Acting Maintenance Approval
FDA greenlights 12-week dosing intervals for maintenance IVIg, easing treatment burdens for chronic CIDP sufferers.

Predictive Wearables Covered by Medicare
Three FDA-cleared gait-and-grip sensor devices now reimbursed, predicting flares 5–7 days early.

Variant CIDP Trial Results
Phase 2 targeted immunomodulation in Lewis-Sumner syndrome shows a 70% response rate, expanding beyond standard IVIg options.

Peripheral Neuropathy Navigator
Foundation for Peripheral Neuropathy’s August 2025 eNews features updated treatment directories, support-group listings, and insurance appeal guides for Texas residents.

Dystonia Clinical Trial Database
CenterWatch’s revamped portal simplifies finding dystonia studies statewide, filtering by subtype and site.

GBS Rehabilitation Guide
Brooks Rehab’s GBS guide offers neurologically trained PT protocols, high-intensity gait training, and virtual-visit options for remote patients.

UT Southwestern Gene Therapy Pipeline
Gray Lab secures NIH funding to develop custom treatments for Childhood-Onset Striatonigral Degeneration, targeting mutations that debilitate children by age 5.

Houston Methodist ALS Platform Trial
Ongoing multi-center studies enroll Texas ALS patients in safety and efficacy assessments of novel therapeutic regimens.

Scottish Rite SNDC Initiative
Dallas’s Scottish Rite for Children and UT Southwestern team award federal grants to pioneer the first SNDC-targeted therapy for this ultra-rare pediatric disorder.

@LoneStarAtaxia (Spinocerebellar Ataxia)
“Joining a local research study gave me my first hope in years—now I walk with less unsteadiness and feel seen.”

@MGWarriorTX (Myasthenia Gravis)
“After fighting insurance denials for complement inhibitors, the new appeal toolkit cut approvals from 8 weeks to 2.”

@GBSSurvivor (Guillain-Barré Syndrome)
“Physical therapy through tele-rehab stopped my relapse and kept me home for family. It changed everything.”

Q: How do I find ataxia clinical trials in Texas?
A: Check the National Ataxia Foundation pipeline and local academic centers like UT Southwestern and Baylor via ClinicalTrials.gov.

Q: Where can I access peer-to-peer CIDP mentorship?
A: Request a match through the GBS-CIDP Foundation’s mentor program—Texas chapters now offer virtual and in-person options.

Q: What telehealth neuro-rehab services exist for rural patients?
A: UT Health Austin’s Rare Disease Tele-Rehab Network provides live virtual PT, OT, and speech therapy, reducing travel by up to 60%.

Issue #6 marks another leap forward for Texas NeuroRare—uniting every rare neurological condition with shared breakthroughs, resources, and community connections. As we head through September, prepare for an exclusive deep dive into Texas’s emerging gene-therapy hub, plus actionable insurance navigation strategies for cutting-edge treatments. Thank you for reading, sharing your stories, and powering this movement.

Neighborly Note & Human Disclaimer:
We’re a small crew doing deep research and sharing real experience—using the best sources we can find, but we’re not perfect (neither is rare disease science or AI!). We are not doctors—so always check with your provider before making health decisions. Spotted an error, have input, or see something confusing? Let us know! All personal stories are anonymous unless you say otherwise. Sponsors never influence our content. We strive for accuracy, but your feedback keeps us honest and helpful. When in doubt, talk to your doc—then come swap stories with us!