A patient with a rare progressive neurodegenerative disorder receiving a personalised experimental antisense oligonucleotide (ASO) treatment developed by the non-profit n-Lorem has reported ...

Diseases that affect the brain and spinal cord can be particularly devastating, and finding new and more effective ways to treat these conditions is an important goal for researchers and clinicians ...

CAMP4 Therapeutics received regulatory clearance in Australia and Argentina to begin a Phase 1/2 trial of CMP-002, its experimental ASO therapy for SYNGAP1-related disorders. The trial will enroll at ...

We are in the golden age of science and technology, advancements in gene editing and ASO therapies are allowing doctors to deliver targeted, and in some cases one-time, interventions for conditions ...

Antisense oligonucleotides (ASOs) have been shown to effectively rescue defects in preclinical Timothy syndrome models, a neurodevelopmental disorder associated with significant morbidity and ...