Five-year-old Kerala girl with SMA in Sharjah begins school journey after life-saving gene therapy

Dubai: Meet five-year-old Aiza Mariyam – a brave Keralite girl from Sharjah who battled the challenges of Spinal Muscular Atrophy (SMA) Type 1 – to take her first steps into the world of learning.
The journey to this day has been anything but ordinary for the young warrior and her parents. Born with SMA Type 1 – a severe genetic condition that affects muscle strength and movement – Aiza’s early life was filled with uncertainties.

Her parents, Naeema and Hyder – both SMA carriers – had already experienced the devastating loss of their first child, a baby boy who succumbed to the same condition just six months after birth. So, when Naeema became pregnant again, she underwent an amniocentesis test in India to detect any genetic abnormalities in the developing foetus. Trusting the medical results, the couple continued the pregnancy. But when Aiza began showing similar symptoms a month after she was born in 2020, the family was heartbroken.

“We were devastated. Everything around us felt dark again,” recalled Naeema, who had left her career as a pharmacist to become Aiza’s full-time caregiver.

Lifesaving treatment

But thanks to swift medical intervention and the Global Managed Access Programme (GMAP), which provides gene therapy to SMA patients around the world, Aiza’s story took a hopeful turn.

Shortly after diagnosis, Aiza’s case was referred to Dr Vivek Mundada, a paediatric neurologist at Medcare Royal Speciality Hospital in Al Qusais. Recognising the urgency of her condition, Dr Vivek applied for the GMAP.

Aiza was just seven months old when she received the life-saving treatment – one of the most expensive drugs in the world – free of cost.
“We were incredibly fortunate to be selected for the GMAP. Dr Vivek and his team have been a blessing,” Naeema said, noting that later the doctor helped Aiza receive Risdiplam – another costly medicine that works in combination with the gene therapy – through a UAE support programme.

‘Nothing short of a miracle’

“With the help of UAE charities and the support of these programmes, Aiza received both treatments at no cost. The generosity of the UAE’s healthcare system and charitable institutions has been nothing short of a miracle,” Hyder, who works as a salesman, underlined.
“Thanks to Dr Vivek’s guidance and Medcare’s team, we received not only the treatment but continuous multidisciplinary care including physiotherapy and regular checkups,” Naeema said, pointing out that Aiza’s follow-ups include physical therapy, neurology, pulmonology, and nutritional checkups.

A new chapter in learning

With a carefully coordinated care plan, Aiza began showing signs of progress. Today, she is full of energy, curiosity and love for learning. She has been enrolled into online learning.
“This is her first structured education experience post-treatment, and she truly enjoys it,” Naeema said with a smile.

The parents opted for virtual learning to protect Aiza’s vulnerable lungs from infections.

“While it’s not a traditional classroom setup, it’s a big step for her to engage with peers and learn activities. We are currently preparing her for big school perhaps next academic year,” said Naeema, noting that Aiza is a bright and curious learner, who loves studying and exploring new things.

Strength in her smile

Proud parents noted that amid the challenges, it was Aiza’s smiling face that gave them strength.
“She is full of life, and her love for learning keeps us and her motivated. We surround her with positivity, celebrate every small milestone, and reassure her that she’s capable of achieving anything that she dreams.”
Over the past five years, they have built their daily routines around Aiza’s therapy and education, aiming to give her a happy and healthy life.

A message of hope

Their message to other families facing SMA is simple: don’t lose hope.

“Medical advances and generous support programmes are changing the story for SMA children. Seek early diagnosis, trust your doctors, and lean on your community. There are people and institutions out there willing to help – you are not alone.”
The family looks forward to a future where Aiza continues to shine.

“Our dream is to see her grow into an independent, educated and kind young woman – someone who knows how strong she truly is. Our greatest goal is to see her live a life full of joy, purpose, and possibility.”