National Neuromuscular Research Institute

10/27/2026

This week, we’re highlighting:
Edios- AG10-501 - A phase 3 Randomized, Multicenter, Double-Blind, Placebo Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial). NCT06563895

Clinical trials help researchers better understand potential new treatments and advance care for people living with Myasthenia Gravis.

Interested in learning more?

Visit clinicaltrials.gov and use ID NCT06563895

If you would like to contact our research team, you can do so at 512-920-0140 opt. 6 OR email us at [email protected].

10/13/2026

This week, we’re highlighting:
80202135MYG3002 (EPIC) - Efficacy and Safety of Nipocalimab vs Efgartigimod for Patients With Generalized Myasthenia Gravis in a Randomized, Open-label, Phase 3b, Interventional Trial Including Within Class Switching From Efgartigimod to Nipocalimab.

Clinical trials help researchers better understand potential new treatments and advance care for people living with Myasthenia Gravis.

Interested in learning more?
Visit clinicaltrials.gov and use ID NCT07217587.

If you would like to contact our research team, you can do so at 512-920-0140 opt. 6 OR email us at [email protected].

10/08/2026

This Dysautonomia Awareness Month, we recognize those living with dysautonomia and the researchers and clinicians working toward better answers.

Symptoms can vary widely and may significantly impact daily life. Increasing awareness is an important step toward earlier recognition, better understanding, and continued research.

09/17/2026

September has been a busy month at NNRI! Check out the latest milestones, study activations, and updates from our clinical research team.

09/04/2026

NNRI has enrolled and dosed the first participant in the Phase 2 clinical trial evaluating apitegromab in adults with FSHD. We are so excited to kick off this study and continue contributing to FSHD research!

08/19/2026

We’re excited to share encouraging news from the Phase 3 ALKIVIA trial!

Argenx has announced positive topline results from the Phase 3 ALKIVIA trial evaluating Vyvgart Hytrulo in adults with autoimmune myositis.

The study met its primary endpoint in the combined immune-mediated necrotizing myopathy (IMNM) and dermatomyositis (DM) population, with improvements observed early and sustained throughout the study.

For people living with these rare neuromuscular conditions, continued research brings hope for a future with more treatment options. We’re encouraged to see progress being made and look forward to learning more as additional results become available.

Read more here: https://argenx.com/news/2026/press-release-3345813.html

The science of co-creation drives our quest to engineer life-changing immunology solutions, the resilient spirit of patients fuels our urgency to deliver them.

08/04/2026

A big thank you to Stephanie, Marisol, and Emil for representing us at Texas Neurological Society's Summer Conference!

TNS was a great opportunity to connect with neurologists, healthcare professionals, and others who share NNRI's commitment to improving care for people living with rare neuromuscular disorders.

Thank you to everyone who visited our booth. We enjoyed the conversations, the chance to share our work, and the opportunity to connect with so many people who are passionate about advancing neuromuscular research.

07/08/2026

Living with a rare neuromuscular disease isn't easy. If these signs sound familiar, it may be time to learn about clinical trial options, especially if your current treatment isn't enough.

07/03/2026

What if your CIDP treatment is helping...but not helping enough?

Many people living with CIDP continue to experience weakness, numbness, fatigue, or symptoms that return before their next treatment. If that sounds familiar, you're not alone.

Clinical research is exploring potential new therapies that may offer another option for people seeking better symptom control.

At NNRI, you'll be cared for by a team dedicated exclusively to rare neuromuscular diseases. Every participant receives specialized, compassionate care while helping advance research that could shape the future of CIDP treatment.

If you're interested in learning whether a clinical trial may be right for you, we'd love to talk.

06/23/2026

Some words from one of our research study participants: "I was 20 and had begun my studies at the University of Arizona when I was diagnosed with a rare and progressive neuromuscular disease... I’m an ambitious person. I’ve tried relentlessly to not let this disease bring me down. I’ve seen a lot of things in my short life, but this has been quite the disaster to say the least. I was a strong man but at the lowest points, I could barely open hands and raise arms. I could barely walk. Picking up a drink or cutting my food with a knife became a progressively more difficult challenge. Every week that passed it got harder to see the future that I had once saw. My medication slowly became less effective over the six five years, though it did keep me going. I’d been asking doctors from across the United States what I should do and what options there were. NO GOOD ANSWERS.

I couldn’t type. I couldn’t work or exercise. I got to a point in my life where I didn’t think there was a way back or be normal again. I didn’t want to face it, but I was out of options. The only treatment I had known for six years wasn’t able to keep me stable and viability was falling apart. From age 20 to early 26 I was skiing downhill without any control. If it wasn’t for Marisol and Dr. Yessar Hussain along with their team I’d be still lost in misery.

The last few months have been a new start to my life. A new me. I’ve never been more grateful to do the simplest of tasks which had been so difficult. Lauren who pokes me every visit doesn’t miss an IV. I’ve been to the various research centers and doctors’ offices in Texas and across the country. This team, their quiet facility, the medicine they have offered me, is unrivaled."