This is Ayah (born on January 15, 2020), a bubbly little princess who likes to smile a lot. Ayah has a rare genetic and degenerative disorder called SMA (Spinal Muscular Atrophy) type 2.
SMA is a rare genetic disease that affects the central nervous system, voluntary skeletal muscle movement, and the peripheral nervous system (the connection between the central nervous system and the rest of the body). As a result, SMA affects basic motor functions such as walking, hand movements and feeding. After progression, the patients lose the ability to swallow, breathe and eventually die.
Since the diagnosis, Ayah has been put on Spinraza, a prescription drug administered as a spinal injection throughout a patient’s life. Spinraza increases the ability of the SMN2 gene (which is a back up of the SMN1 gene-the one that Ayah is missing) to produce SMN protein which in turn improves motor functions and survival.
Zolgensma is a new one time gene therapy treatment for kids under the age of 2. It REPLACES the missing SMN1 gene with a new, working copy that helps motor neuron cells work properly and survive but it’s currently the most expensive drug in the world at $2.1 million. It is NOT a cure since it does not reverse damage already caused by SMA before treatment. However, with supportive therapy, it has by far shown the best results. It is recommended that the earlier the patient receives the treatment, the better.
We would like to invite you to help Ayah’s family, friends and network create awareness around this fundraising by sending us your prayers, positive thoughts, contribute financially and/or share this invitation with more networks so that we may reach as many helpful souls as possible. Every single contribution means the world to us.
We hope to get Ayah treated as soon as we raise all the funds needed. $2.1 million will be used to buy the drug zolgensma. The remaining $300,000 and any additional donations will be used for treatment at Boston Children’s Hospital, travel and accommodation during our stay in the USA. Thank you so much for your kind donations!!