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It was a pleasure to welcome the next generation of bright young minds to our offices for this year’s  ! 🔬🧬From research...
04/24/2026

It was a pleasure to welcome the next generation of bright young minds to our offices for this year’s ! 🔬🧬

From research and development to patient services and beyond, they were able to see firsthand how our teams collaborate with the shared goal of serving rare disease communities.

It was a meaningful reminder that when we come together to make a difference.

This  , we are proud to support the World Health Organization (WHO)’s Together for Health campaign, celebrating the powe...
04/07/2026

This , we are proud to support the World Health Organization (WHO)’s Together for Health campaign, celebrating the power of collaboration in driving meaningful scientific advancements in healthcare.

At NS Pharma, our relationships with patients, caregivers, advocacy groups, and healthcare professionals help us address unmet needs, understand the patient journey, and drive meaningful innovation.

The dystrophin gene is made up of 79 segments known as exons. Exon-skipping therapies are designed to skip over a specif...
03/30/2026

The dystrophin gene is made up of 79 segments known as exons. Exon-skipping therapies are designed to skip over a specific exon, which may help the body work around certain genetic mutations.

Genetic testing can help determine which exons are impacted and whether exon-skipping could be a potential approach.

Speak with your healthcare provider about testing options and learn more about DMD here: https://ow.ly/emjI50YB61H

We’re looking forward to spending time at this upcoming community event and stepping into meaningful conversations aroun...
03/27/2026

We’re looking forward to spending time at this upcoming community event and stepping into meaningful conversations around rare disease care. 💙

Opportunities like this remind us that progress is not only measured in data, but also in dialogue. Listening to lived experiences, sharing perspectives, and continuing to learn together all play a role in moving care forward.

If you will be attending, we're looking forward to connecting and continuing the conversation.

Learn more about the event: https://ow.ly/KaGF50YzCzX

03/25/2026

When your immune system shifts from defense to offense, your body might feel like it’s under attack.

Eosinophilic Granulomatosis with Polyangiitis (EGPA) is a rare disease, characterized by a mix of symptoms including asthma, sinusitis, skin rashes, and nerve pain - which makes it challenging to diagnose due to its rarity and symptom overlap with other diseases.

EGPA patients often have higher than normal levels of an immune cell called eosinophils, and the overactivity of these cells causes inflammation of small blood vessels. Immune activity is regulated by signaling pathways that involve enzymes like JAK1. JAK1 inhibitors are designed to turn down the activity of immune cells, and are being studied as an approach to address inflammation associated with EGPA.

Learn how our NS-229 JAK1 inhibitor is being evaluated in a Phase 2 global clinical trial:
https://ow.ly/mngi50YyAPe

03/24/2026

Duchenne muscular dystrophy is part of your story, not the whole story.

Brantley was just 4 years old when his family began noticing early signs of Duchenne. Doctors later discovered he had low levels of dystrophin - the vital protein that helps strengthen and protect muscles. Without functional dystrophin, his family worried whether he could continue the activities he loves, like woodworking.

Exon-skipping therapies help the body produce a shortened but usable form of dystrophin that slows the progression of muscle degeneration, so that patients like Bradley continue to pursue their passions and dreams.

Learn more about NS Pharma’s work on DMD treatments: https://ow.ly/xenj50Yy30q

03/23/2026

Duchenne muscular dystrophy is part of your story, not the whole story.

Bradley was just 4 years old when his family began noticing early signs of Duchenne. Doctors later discovered he had low levels of dystrophin - the vital protein that helps strengthen and protect muscles. Without functional dystrophin, his family worried whether he could continue the activities he loves, like woodworking.

Exon-skipping therapies help the body produce a shortened but usable form of dystrophin that slows the progression of muscle degeneration, so that patients like Bradley continue to pursue their passions and dreams.

Learn more about NS Pharma’s work on DMD treatments: https://ow.ly/FMJm50YxtMZ

We are proud to support and attend the 2026 Brian McKenna TetraSki Express National Championship on March 27 at Powder M...
03/20/2026

We are proud to support and attend the 2026 Brian McKenna TetraSki Express National Championship on March 27 at Powder Mountain Resort in Eden, Utah.

This extraordinary event highlights the power of adaptive technology, athleticism, and resilience as athletes with complex physical injury or illness compete in TetraSki and Snow’Kart downhill racing

What a celebration of inclusion, innovation, and the human spirit! We're honored to stand alongside a community that continues to redefine what is possible and to support initiatives that promote equity and opportunity in adaptive sports.

Learn more about the event: https://ow.ly/Qp6p50YwMcT

We’re glad to be part of this upcoming event and to continue showing up for the rare disease community. 💙Moments like th...
03/18/2026

We’re glad to be part of this upcoming event and to continue showing up for the rare disease community. 💙

Moments like these are an opportunity to pause, reflect on progress, and stay grounded in what matters most. Every conversation and every connection helps shape how we think about care, access, and the future of treatment.

We look forward to seeing you there!

Learn more about the event: https://ow.ly/maS550YvHtO

Thank you to Project Alive and the Hunter syndrome community for a meaningful Building a Future Together Conference. 💙Sp...
03/16/2026

Thank you to Project Alive and the Hunter syndrome community for a meaningful Building a Future Together Conference. 💙

Spending time with families, advocates, and community leaders in Costa Mesa was a powerful reminder of the strength and dedication that drives progress in rare disease care. The conversations, insights, and shared experiences continue to shape how we listen, learn, and move research forward.

We are grateful to everyone who welcomed us into these discussions and look forward to continuing our work alongside this incredible community.

We’re looking forward to attending the Akari Foundation Workshop on March 14 to 15, 2026. 💙Workshops like this create me...
03/12/2026

We’re looking forward to attending the Akari Foundation Workshop on March 14 to 15, 2026. 💙

Workshops like this create meaningful space for focused discussion, shared learning, and continued momentum in rare disease research and care. Spending time in these environments allows us to stay connected to the evolving needs of the community and the science shaping the future.

We appreciate the Akari Foundation’s dedication to bringing people together around a shared purpose and look forward to seeing you there!

Learn more about the workshop: https://ow.ly/AzGa50YsKmi

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