Countdown Has Begun: 2-Year-Old Girl with SMA Faces a Life Permanently Altered If She Misses This Treatment. Parents Urgently Appeal for Public Support to Raise RM520,000 in Medical Costs

At this age, many children are learning to stand unsteadily, laughing as they run into their parents’ arms.
But for this 2-year-old girl, standing independently is still impossible.

It is not because she is not trying. She is battling a rare genetic condition Spinal Muscular Atrophy (SMA) Type 2 which is gradually weakening her muscles, day by day.

Hailing from Kampar, Perak, Nur Fathiah Aisyah urgently requires RM520,000 in medical expenses. This is not an illness that can be delayed. Once the critical treatment window is missed, lost physical functions may no longer be recoverable.

【Treatment for SMA cannot be administered at any time. Missing the appropriate intervention window may lead to irreversible loss of physical function】

Nur Fathiah Aisyah appeared healthy at birth. However, by the age of one, her father began to notice that she was still unable to sit independently and could only move by crawling slowly on her stomach. Although the family raised their concerns during routine medical visits, they were repeatedly reassured that “nothing was wrong.”

Unconvinced, her father insisted on further medical evaluation. After being referred to specialist care and undergoing multiple assessments and genetic testing, doctors finally confirmed in October last year that she was diagnosed with Spinal Muscular Atrophy Type 2 (SMA Type 2).

Doctors explained that SMA is a neuromuscular disease that progressively affects muscle strength and mobility. Without timely intervention, the condition will continue to deteriorate. While treatment options do exist, they are extremely costly and not suitable for every patient.

During this difficult period, the parents learned of an international gene therapy option for SMA patients. One such treatment, Vesemnogene Lantuparvovec, requires only a single injection and has the potential to significantly improve their daughter’s health and quality of life.

Following medical evaluation and confirmation, doctors determined that this one-time gene therapy is effective for her condition. However, the treatment must be administered within a specific therapeutic window. Nur Fathiah Aisyah has been scheduled to receive the treatment on 26 January, which means she must depart by 25 January.

The cost of this treatment amounts to RM520,000 an amount far beyond the family’s means after exhausting their savings and taking on debt in their search for treatment.

“We have no other options left. This gene therapy is our only hope. We are willing to bring our child to Tzu Chi Hospital in Indonesia for treatment. All we wish for is for her to be able to stand, walk, and live a healthy life like other children,” said the parents.

Nur Fathiah Aisyah’s father, Muhammad Fitry bin Mohd Hanafi (31), is an employee of Tenaga Nasional Berhad with a basic salary of approximately RM2,145. Her mother, Nur Farah Atika binti Mohd Khamis (30), is a full-time housewife who provides round-the-clock care and rehabilitation for their daughter.

While the family can manage airfare and basic living expenses, they are unable to bear the RM520,000 cost of the one-time gene therapy on their own.

We sincerely appeal to the public to extend a helping hand and stand with us in preserving this young child’s chance to walk in the future.

After review, One Hope Charity has approved this case and resolved to assist in fundraising the required medical expenses. The family has entrusted One Hope Charity with full responsibility for organizing and managing the fundraising campaign. For this case, the Foundation will allocate RM50,000 from the Spinal Muscular Atrophy (SMA) Medical Reserve Fund.

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