Wow! Something amazing!
New Data Released
In January 2016, AveXis reported interim data through the December 31, 2015 time period from the ongoing Phase 1 trial of AVXS-101 in SMA Type 1. The data reported in this release reflects that same dataset and time period. AveXis reported that AVXS-101 appears to have a favorable safety profile and appears to be generally well tolerated in patients studied as of December 31, 2015. As of that date, there had been a total of 10 serious adverse events (SAEs) reported; two of these were determined to be related to therapy and involved clinically asymptomatic, elevated liver function enzymes. Both cases have resolved.
As of December 31, 2015, no patient in either dosing cohort had experienced an “event.” An event is defined as death, or until a patient requires at least 16 hours per day of ventilation support for breathing for 14 consecutive days in the absence of an acute reversible illness, or perioperatively. The median event-free age of all 15 patients was 11.8 months, with the oldest patient at 25.8 months of age. In Cohort 1, all 3 patients were over 20 months of age and event-free. In Cohort 2, the first 6 patients to be treated were greater than 10.5 months of age and event-free.
As of December 31, 2015, mean increases of 6.0 points and 18.0 points in CHOP-INTEND scores were observed in Cohort 1 and Cohort 2, respectively. The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) is a test developed to measure motor skills of patients with SMA Type 1, and other motor milestone development surveys and tests.
Upcoming Clinical Trials
Enrollment for this Phase 1 trial has recently been completed. AveXis plans to initiate Phase 2 trial of systemic delivery in the first half of 2017. AveXis has also announced plans for a Phase 1 trial testing a second approach, CSF delivery, in individuals with SMA type II. This trial is scheduled to begin in the second half of 2016.
Full text: http://www.curesma.org/news/avexis-new-data-phase-1.html?referrer=https://www.facebook.com/
My name is Monika Lemeshonok, I am painter from Belarus. From born I have rare disease of the spinal cord - Spinal Muscular Atrophy Type 2. From this disease, I can’t walk and move, I'm in a wheelchair. But I love life and around the world. I express my love for life through painting artwork, I have art exhibitions throughout Europe and many art lovers appreciate my works.
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