NS Pharma is working to have an important impact on the lives of patients with rare diseases. Our therapeutic areas encompass neurological diseases and inflammatory diseases.
Harnessing the power of innovative science can help bring new treatments to patients with rare diseases. NS Pharma is developing therapies through exon-skipping, cell therapy and JAK1 inhibition technology.
NS Pharma is working to have an important impact on the lives of patients with rare diseases. Our therapeutic areas encompass neurological diseases and inflammatory diseases.
Harnessing the power of innovative science can help bring new treatments to patients with rare diseases. NS Pharma is developing therapies through exon-skipping, cell therapy and JAK1 inhibition technology.
Press Release / October 14, 2025
Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress
Press Release / October 14, 2025
Brogidirsen (NS-089/NCNP-02) 3.5 Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2025 World Muscle Society Congress
Indiana families, join us for the Best Friendsgiving Ever: a special gathering for those living with #Duchenne!
📅 Sat, Nov 22
🕓 4–7 PM
📍 Local 416 Firefighters Union Hall, Indianapolis.
Food, fun & connection await!
Register: https://ow.ly/ekGq50XuzeC
#FamilySupport https://x.com/NSPharmaInc/status/1991884784369385598/photo/1
Today we celebrate International Children’s Day, honoring the strength, courage & potential of children everywhere.
At #NSPharma, we’re driven by a commitment to improve the lives of children & families affected by rare diseases.
#HopeInAction https://x.com/NSPharmaInc/status/1991522448487944490/photo/1
Did you know the dystrophin gene has 79 exons?
When one is missing, Duchenne Muscular Dystrophy can occur. Exon skipping therapies “skip over” faulty exons, helping the body produce functional dystrophin.
Learn more: https://ow.ly/joG350XtYcC
#DuchenneAwareness #ExonSkipping https://x.com/NSPharmaInc/status/1991159550183907741/video/1
We’re proud to sponsor Austin’s Hope and their mission to support families impacted by Duchenne.
Join the Night of Hope Gala on Nov 22 in Marysville, OH for an evening of music, connection & giving back.
Learn more: https://austinshope.org
#AustinsHope #Duchenne #NSPharma https://x.com/NSPharmaInc/status/1990797530041290965/photo/1
Welcome Audra Lapati to #NSPharma as Area Business Manager!
With 20+ years in strategic account management across neurology, oncology & rare diseases, including roles at UCB, Takeda & Nutricia, she brings deep expertise to our team. We’re thrilled to have you!
#NewHire https://x.com/NSPharmaInc/status/1990435367749644518/photo/1
We’re excited to attend the #CureDuchenne Workshop in Boston!
This event unites families, clinicians & researchers to improve outcomes for those living with Duchenne.
📅 November 15
🕘 8:00 AM – 4:00 PM
📍The Westin Waltham
Learn more & register: https://ow.ly/kjq750XpZkL https://x.com/NSPharmaInc/status/1989348045683560634/photo/1
Leber congenital amaurosis is caused by mutations in the GUCY2D gene, disrupting retinal function.
We're partnering with Atsena Therapeutics to advance ATSN-101, an investigational #GeneTherapy aiming to restore vision.
Learn more: https://ow.ly/GCzY50XpZ2s
#LCA1 #RareDisease https://x.com/NSPharmaInc/status/1988623324562268474/photo/1
