ALL WE NEED IS ZOLGENSMA CAN SOMEONE HELP MY BABY

We, Miriam Manalo (Mother) and Kalid Maher Al Raee (Father) from the Philippines are appealing to all of you and knocking on your kind hearts to help us raise funds for our daughter, Lilah Emilia, who is diagnosed with Spinal Muscular Atrophy (SMA).

We already lost our first born last October 8, 2019 as she was diagnosed with SMA Type 1. Our first angel’s name was Layla Elleina Al Raee, she was only 8 months old.

We are blessed to be given a chance to have a second daughter born last December 7, 2020. She’s pictured above, our beautiful Lilah Emilia Al Raee, 6 months old, and unfortunately been diagnosed with the same disease as our first born.

Please help our family raise the funding needed for Lilah’s immediate treatment and associated travel expenses. There are few treatments for this illness such as Zolgensma, Sprinraza, and Evrysdi but unfortunately not available in our country, the Philippines. We still need to bring our daughter to the US for treatment. The treatment alone already costs millions of dollars.

We are so heartbroken but as parents who have already lost a child, we don’t want to lose another one. We trust and believe that God will help us and provide us a miracle through the help of your donations as well.

We are trying to raise $2.5 million for Lilah's rare medical condition, so any help you can give will go a long way.

We know how hard we’re all coping because of the pandemic, but if you're able to spare some extra funds, please help our daughter to survive.

Please share our story and if you can also include us in your prayers, that means a lot.

For those who are not yet familiar with SMA, here’s a brief about the disease:

(Spinal Muscular Atrophy) is a genetic (inherited) neuromuscular disease that causes muscles to become weak and waste away. People with SMA lose a specific type of nerve cell in the spinal cord (called motor neurons) that control muscle movement. Without these motor neurons, muscles don’t receive nerve signals that make muscles move. The word atrophy is a medical term that means smaller. With SMA, certain muscles become smaller and weaker due to lack of use. Type 1 (severe): About 60% of people with SMA have type 1 , also called Werdnig-Hoffman disease. Symptoms appear at birth or within an infant’s first six months of life. Infants with type 1 SMA have difficulty swallowing and sucking. They don’t meet typical milestones like holding up their heads or sitting. As muscles continue to weaken, children become more prone to respiratory infections and collapsed lungs (pneumothorax). Most children with type 1 SMA die before their second birthday.

While 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 need to raise enough funds $400,000 to $2.5M to cover the costs of the gene-therapy treatment (Zolgensma), Spinraza, or Risdiplam and additional expenses like travel, consultation fee and accommodation during her treatment abroad. By giving Lilah this treatment, we hope to give her an almost normal childhood without any major obstacle. We are in a race against time to achieve our goal since this treatment is currently only approved for children under 2 years old.

Thank you so much for your kind donations

Philippine doctors that handle the case of Lilah Emilia

Pediatrician - Dr. Maritess Henson-Tayag
Neurologist - Dr Christina Urbi
Neurophysiologist - Dr Kaye Banzali
Geneticist - Dr. April Grace