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We’re excited to be in Chicago with our friends from CureDuchenne for another special community dinner!This evening is d...
11/07/2025

We’re excited to be in Chicago with our friends from CureDuchenne for another special community dinner!

This evening is designed for caregivers and families impacted by Duchenne and Becker muscular dystrophies. These dinners are always a wonderful opportunity to connect, share stories, and find support together.

🗓️ Date: November 8
⏰ Time: 6:00 PM – 9:00 PM
📍 Location: Chicago Prime Steakhouse, 1444 E Algonquin Rd, Schaumburg, IL 60173

👉 Reserve your spot today:
https://ow.ly/A4Mt50XnBNa

A big thank you to everyone who visited our poster presentation at the AANEM Annual Meeting last week, and to our brilli...
11/06/2025

A big thank you to everyone who visited our poster presentation at the AANEM Annual Meeting last week, and to our brilliant colleagues Mara Wood, Michelle Previtera, and Denise Quigley for sharing their expertise and insights!

If you missed the presentation, you can view the poster here: https://ow.ly/q98550XnBEi

Progress in rare diseases happens through collaboration, curiosity, and connection - and brought all three to life. Thanks to American Association of Neuromuscular & Electrodiagnostic Medicine - AANEM for hosting such a meaningful event.

We look forward to the next one!

We’re very proud to sponsor Hope for Gus and their Everest to End Duchenne trek! 🏔️This incredible journey to Everest Ba...
11/05/2025

We’re very proud to sponsor Hope for Gus and their Everest to End Duchenne trek! 🏔️

This incredible journey to Everest Base Camp unites families, advocates, and adventurers to raise awareness and funds for Duchenne muscular dystrophy (DMD) research. Each step taken reflects the determination of those living with Duchenne, and our shared commitment to finding a cure.

Learn more and support this inspiring mission: https://everesttoendduchenne.org

We are conducting a Phase 2 clinical study evaluating NS-229, an investigational JAK1 inhibitor, to learn more about pot...
10/31/2025

We are conducting a Phase 2 clinical study evaluating NS-229, an investigational JAK1 inhibitor, to learn more about potential new treatment options for adults with EGPA.

Every advancement in research begins with those willing to take part.

Learn more and see if you are eligible to join:
https://ow.ly/UUAW50XkWaw

🌟 We’re proud to sponsor the Jett Foundation’s final Family Workshop of 2025, uniting families, advocates, and experts f...
10/29/2025

🌟 We’re proud to sponsor the Jett Foundation’s final Family Workshop of 2025, uniting families, advocates, and experts from the Duchenne community for a day of learning, connection, and empowerment.

At NS Pharma, we’re honored to support Jett Foundation’s mission to strengthen the Duchenne community through education, advocacy, and shared experience, driving progress and hope for all those impacted. 💙

📍 Sheraton Norfolk Waterside Hotel – Norfolk, VA
📅 Saturday, November 8, 2025 | 10:00 AM – 4:30 PM

This in-person workshop will feature educational sessions, community resources, and opportunities to connect directly with clinicians and industry partners dedicated to improving lives impacted by Duchenne.

🔗 Register to join the event: https://jettfoundation.formstack.com/forms/norfolk_va_family_workshop_registration

We're delighted to be presenting some exciting new data at the American Association of Neuromuscular & Electrodiagnostic...
10/27/2025

We're delighted to be presenting some exciting new data at the American Association of Neuromuscular & Electrodiagnostic Medicine - AANEM Annual Meeting this week!📊

Don't miss our poster presentation examining exon-skipping therapy persistence at:
🔹Session I: Thursday, 10/30 at 6:15 PM
🔹Session III: Friday, 10/31 at 2:45 PM

Our colleagues Mara Wood and Denise Quigley will be there to discuss the findings and their impact on the Duchenne community.

We look forward to connecting with you in San Francisco!

We’re excited to attend the 10th Annual Utah Program for Inherited Neuromuscular Disorders (UPIN) Family Conference!On O...
10/24/2025

We’re excited to attend the 10th Annual Utah Program for Inherited Neuromuscular Disorders (UPIN) Family Conference!

On October 25, 2025, we’ll join patients, families, and clinicians at the Loveland Living Planet Aquarium for a day of learning, connection, and community and, of course, some time exploring the aquarium - a favorite for the kids! 🐠

Since 2015, UPIN has united those impacted by neuromuscular disorders through education, research, and collaboration. This year’s event will feature engaging speakers, family-led discussions, and fun activities for all ages.

📍 Loveland Living Planet Aquarium
📅 Saturday, October 25 | 9 AM–3 PM

Register here to join us: https://ow.ly/Y1o150XgP1U

Hunter Syndrome (MPS II) is a rare, life-limiting genetic disorder that primarily affects boys. It occurs when the body ...
10/23/2025

Hunter Syndrome (MPS II) is a rare, life-limiting genetic disorder that primarily affects boys. It occurs when the body is missing a crucial enzyme responsible for breaking down and clearing out cellular waste, leading to progressive damage over time.

During Hunter Syndrome Awareness Week (October 19–25), we stand with the families and advocates working tirelessly to raise awareness and support continued research into this devastating condition.

At NS Pharma, we remain committed to advancing science for rare diseases like MPS II, helping bring hope to those impacted. 💜

🎥 Learn more about Hunter Syndrome: https://ow.ly/mTC650XgOR7

The American Association of Neuromuscular & Electrodiagnostic Medicine - AANEM Annual Meeting kicks off next week and we...
10/22/2025

The American Association of Neuromuscular & Electrodiagnostic Medicine - AANEM Annual Meeting kicks off next week and we’re excited to join leading experts and innovators driving progress in neuromuscular medicine. 🌉

This premier gathering unites physicians, technologists, advanced practice providers, and healthcare professionals from around the world to explore the latest in NM, MSK, EDX, and NMUS, plus exchange insights that advance patient care!

📍 Hilton San Francisco Union Square, San Francisco
📅 October 29 – November 1, 2025

Don’t miss our poster presentation ( #294) on Thursday and Friday, where we’ll share new insights on exon-skipping and its potential for the Duchenne community!

📊 Happy World Statistics Day!At NS Pharma, data isn’t just numbers, it’s what guides every breakthrough in our mission t...
10/20/2025

📊 Happy World Statistics Day!

At NS Pharma, data isn’t just numbers, it’s what guides every breakthrough in our mission to help those living with rare diseases.

Our analysts turn complexity into clarity, ensuring every decision in research and clinical development is grounded in evidence. Their work helps transform uncertainty into progress, and shows that when data leads, discoveries follow!

Did you know that October is Hunter Syndrome Awareness Month?💜Hunter Syndrome, also known as MPS II,  is a rare, progres...
10/17/2025

Did you know that October is Hunter Syndrome Awareness Month?💜

Hunter Syndrome, also known as MPS II, is a rare, progressive genetic disorder that primarily affects boys and touches every part of life.

At NS Pharma, we’re proud to stand with the MPS community and are advancing research through our investigational gene therapy, RGX-121, developed in partnership with REGENXBIO. While still investigational, it represents hope for families affected by this rare disease.

Learn more about the condition and how you can help: https://ow.ly/UC1o50XbIIQ

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