RESEARCHERS CORRELATE SPINAL MUSCULAR ATROPHY DISEASE EXPRESSION WITH HAPLOTYPES

STRASBURG, PA- A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy (SMA) within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes and SMN2 copy number with disease severity. SMA is a devastating genetic disease that affects the motor neurons that control… Att fortsätt läsa

New Method of NUSINERSEN/Spinraza Delivery with intrathecal catheter (SIC) system, give more SMA Patients Access and reduce at same time costs five- to ten-fold

and this is shown by a new study from Clinc for Special Children in Strasburg the method has already been developed and tested there. STRASBURG, PA- A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system… Att fortsätt läsa

Nusinersen/Spinraza shows the effectiveness in children older than 7 months

This show a new Cohort study. That is no secret anymore. Children and adults benefit from Spinraza, recent studies show this again We treated 33 children ranging in age from 8.3 to 113.1 months between December 2016 and May 2017. All patients were alive and were continuing treatment at M6…. Att fortsätt läsa