Saudi Arabia sees 200-250 new SMA births a year, yet a legacy cohort of 800-1,200 GCC patients remains ineligible for gene therapy entirely.

Spinal muscular atrophy incidence in the GCC runs 1 in 6,000 to 8,000 live births versus roughly 1 in 10,000 globally, elevated by consanguinity increasing the odds of homozygous SMN1 deletion. Saudi Arabia alone sees an estimated 200-250 new SMA births annually against roughly 250,000 total births, with the UAE contributing 30-35 and Qatar 20-25, a combined GCC total near 300-350 new cases a year. Newborn screening is converting these from symptomatic diagnoses to pre-symptomatic identification at very different rates by country: an estimated 90% in Saudi Arabia, 85% in the UAE, 75% in Qatar, and below 50% across the remaining states.

The clinical stakes of that screening gap are large. Pre-screening SMA Type 1 median survival ran near 2 years; with newborn-screening identification and immediate Zolgensma treatment, survival in the NURTURE cohort approaches that of unaffected children, and pre-symptomatic infants at centres including KAMC, KFSH&RC, Sidra Medicine, and SKMC are reaching normal motor milestones. Alongside this pre-symptomatic cohort sits a distinct unmet-need population: an estimated 800-1,200 GCC SMA Type 2/3 patients diagnosed before screening existed, now teenagers and adults with established motor disability. These patients are ineligible for gene therapy on age and weight criteria and depend on chronic nusinersen or risdiplam, or remain untreated, while physical therapy and respiratory support outside tertiary centres stays limited.

Screening is solving the newborn cohort; the legacy cohort still needs an answer.

Five questions this report answers:

Q1 - How does GCC newborn-screening coverage vary by country, and what does that mean for the near-term Zolgensma-eligible population?

Q2 - What is the size and treatment status of the pre-NBS Type 2/3 adult SMA population across the GCC?

Q3 - What non-drug infrastructure, like physical therapy and respiratory support, constrains SMA outcomes outside GCC tertiary centres?

Q4 - How many new SMA cases does the GCC see annually, and how is that split by country?

Q5 - Which GCC centres, including KAMC and Sidra Medicine, are delivering pre-symptomatic gene therapy today?

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#SMA #SpinalMuscularAtrophy #GCC #NewbornScreening #RareDisease #DiseaseLandscape

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Mike || Global Pharma Commercial Marketing Head

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