New Treatment for ALS Gives Hope to Patients and Families
4 minutes
Meet Rescue 7: Firefighters Serving Patients
with Luke Rosen, Raena and Frank Vrtochnick
Restoration of GBE1 Protein Activity with an ASO: Impact of a Common Sequencing Error on Drug Discovery
Let’s Discuss Science [...]
n-Lorem Foundation Publishes Study Showing Targeted ASO Therapy Restores GBE1 Protein in APBD Patient Cells
Research, published in Nucleic [...]
Lesson 17 – The Science of ASO Design and Screening Using the FDA Guidance for Nano-rare Patients
Lessons in Antisense [...]
Hope for Kinsley: Facing TUBB4A with Courage and Care
with Sarah and Ryan Marta
Lesson 16 – PS ASO-induced PS ASO-RNP Aggregates
Lessons in Antisense [...]
Lesson 15 – The Mechanisms of PS ASO-induced Cytotoxicity
Lessons in Antisense [...]
New Insights into Genetically Caused Developmental Delays
Emerging [...]
