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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

We're excited to be attending the Project Alive Building a Future Together Conference on March 14th-15th, 2026. 💙This ga...
03/11/2026

We're excited to be attending the Project Alive Building a Future Together Conference on March 14th-15th, 2026. 💙

This gathering is an important opportunity to listen, learn, and engage with families and advocates in the Hunter syndrome community. Time spent together in conversation helps strengthen understanding and reinforces the shared commitment to improving care and advancing research.

We're grateful for the dedication of Project Alive and the families who continue to lead with courage and collaboration. We look forward to being part of the experience.

Learn more about the conference: https://ow.ly/5GMK50YsfZC

Our team is onsite at MDA 2026 and looking forward to engaging with our community! On Sunday and Monday, we’ll be presen...
03/09/2026

Our team is onsite at MDA 2026 and looking forward to engaging with our community! On Sunday and Monday, we’ll be presenting three additional posters highlighting real world evidence. Stop by posters 146 M, 168 M & 57 S to learn more!

Please join us in welcoming Cassandra Kaufhold to NS Pharma’s Medical Affairs & Pharmacovigilance team as a Medical Scie...
03/06/2026

Please join us in welcoming Cassandra Kaufhold to NS Pharma’s Medical Affairs & Pharmacovigilance team as a Medical Science Liaison! 👋

Cassandra brings more than seven years of translational preclinical experience in neurodegenerative and rare diseases, with a strong focus on clinical translation and novel therapeutic targets. She holds a Bachelor of Science from the University of Pittsburgh and a Ph.D. in Neuroscience from Texas A&M University College of Medicine.

She joins us from Texas A&M, where she spent the past four years as a graduate research assistant leading multiple translational projects focused on Parkinson’s disease, inflammatory bowel disease, and the gut brain axis. Cassandra is a selected scholar of the Neural Gut Immune Axis Consortium and a first author on multiple published manuscripts and reviews.

Outside of work, Cassandra enjoys staying active, caring for her house plants, experimenting with new recipes, and exploring Houston’s coffee scene. ☕

Welcome to the team, Cassandra!

Looking for support? You're not alone. 💙Families navigating rare diseases often face challenges that go beyond medical c...
03/04/2026

Looking for support? You're not alone. 💙

Families navigating rare diseases often face challenges that go beyond medical care. Access to housing support, transportation, financial assistance, food resources, and community programs can make a meaningful difference.

The Little Hercules Foundation offers a searchable tool to help families find local support services quickly and privately using their ZIP code - designed specifically for the rare disease community.

We are proud to partner with organizations like Little Hercules that provide practical tools to help families access the resources they need.

Explore the resource here: https://ow.ly/H9AC50YoYVi

This weekend we’re headed to Orlando for the 2026 MDA Clinical & Scientific Conference, where we’ll be sharing new data ...
03/02/2026

This weekend we’re headed to Orlando for the 2026 MDA Clinical & Scientific Conference, where we’ll be sharing new data from our ongoing research in Duchenne muscular dystrophy.

This year, we will be presenting five abstracts, including findings from a long-term open label extension study evaluating brogidirsen (NS-089/NCNP-02), an investigational therapy for DMD patients with dystrophin gene mutations amenable to exon 44 skipping.

Stop by booth #213! We look forward to discussing these data, exchanging perspectives, and connecting with clinicians, researchers, and advocates who share a commitment to advancing neuromuscular disease research.

Today, on RareDiseaseDay, we’re showing our stripes in support of the rare disease community. Today and every day we are...
02/27/2026

Today, on RareDiseaseDay, we’re showing our stripes in support of the rare disease community. Today and every day we are inspired by the resilience of patients, caregivers, and families, and remain committed to advocating for earlier diagnosis, improved access, and unwavering support across the rare disease community

Learn more about National Organization for Rare Disorders’s Show Your Stripes campaign and ways to get involved: https://rarediseases.org/rare-disease-day/

02/23/2026

Living with Duchenne muscular dystrophy is a journey of strength and resilience. As approaches, we’re honoring the wider rare disease community and the courage behind every story.

Visit https://rarediseases.org/rare-disease-day/ to learn more and join us in raising awareness.

Dallas, we’re headed your way. 💙We are excited to take part in the upcoming CureDuchenne Workshop and spend the day imme...
02/20/2026

Dallas, we’re headed your way. 💙

We are excited to take part in the upcoming CureDuchenne Workshop and spend the day immersed in meaningful dialogue around Duchenne muscular dystrophy. These gatherings create space for real conversations about care, research, and the lived experiences of families navigating Duchenne.

We value the opportunity to be present, to learn, and to support a community that continues to inspire progress.

Learn more about the workshop: https://ow.ly/V9FX50YiHJv

02/18/2026

With approaching on 2/28, we want to thank the families who’ve allowed us to be a part of their Duchenne journey. Their stories remind us why awareness and education are essential.

Explore their Duchenne journeys: https://ow.ly/S7vL50Yhu5C

What an incredible experience at WORLDSymposium 2026!We were grateful to see well over 70 attendees join our session and...
02/13/2026

What an incredible experience at WORLDSymposium 2026!

We were grateful to see well over 70 attendees join our session and take part in a meaningful conversation around MPS II. A special thank you to Drs. Hoffman, Burton, and Jalazo for delivering an outstanding presentation that provided a thoughtful overview of the disease, insights into the evolving paradigm of care, and powerful reflections drawn from personal and clinical experience.

The discussion and Q&A were so engaging that we had to bring the session to a close after running over our allotted time, which speaks to the level of interest and collaboration in the room.

Thank you to everyone who attended, asked questions, and helped make this such a memorable and impactful session. We look forward to continuing these important conversations.

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