"Spinal Muscular Atrophy (SMA) is a motor neuron disease. The motor neurons affect the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. It is a relatively common "rare disorder": approximately 1 in 6000 babies born are affected, and about 1 in 40 people are genetic carriers." Currently, there is no cure. ISIS Pharmaceuticals, Inc. is currently holding clinical trials for the drug ISIS-SMNrx for children who meet the criteria.
Our daughter, Harper Olivia, was diagnosed at 6 months of age, but doesn't qualify for the trial. If Harper doesn't get treatment, she will continue to regress. Time is precious for children with SMA and waiting for FDA approval could take years. We simply don't have time to wait. As her parents, we are responsible for fighting for her. Many other familes are in the same situation as we are. Therefore, we ask not only for Harper to be allowed compassionate use, but for other families as well.
If Harper and other affected children who don't qualify for the trial are not allowed compassionate use, they can lose the battle against SMA. Every child with SMA should be able to receive this potentially life changing treatment. Please sign this petition in the fight against SMA! Let ISIS Pharmaceuticals, Inc. hear your voice! By signing, you are asking ISIS to grant compassionate use for each child, whose parents want the opportunity, to try ISIS-SMNrx now!
- Director, Communications and Advocacy Relations, ISIS Pharmaceuticals
Kristina (Lemonidis) Bowyer
- founder, CEO & chairman of the board of directors - ISIS Pharmaceuticals, Inc.
Stanley T. Crooke, Ph.D., M.D.
Grant Compassionate Use of ISIS-SMNrx to Harper Olivia Singh and other children who don't qualify for clinical trial.
Holly Singh started this petition with a single signature, and now has 13,420 supporters. Start a petition today to change something you care about.