Abu Dhabi has reached a significant milestone in global healthcare by administering the ITVISMA gene therapy to a patient suffering from spinal muscular atrophy (SMA). This marks a pivotal advancement in precision medicine and genomics-based healthcare.
On December 28, 2025, Sheikh Khalifa Medical City successfully delivered ITVISMA, a one-time gene therapy for SMA. This groundbreaking treatment was given under the supervision of the Department of Health, Abu Dhabi, following its regulatory approval on November 25, 2025. Consequently, the UAE has become the second country, after the United States, to approve ITVISMA for clinical application.
ITVISMA is an innovative gene therapy developed by Novartis that specifically targets the genetic cause of spinal muscular atrophy. Here are its key features:
Spinal muscular atrophy is a rare genetic neuromuscular disorder characterized by:
SMA stems from mutations in the SMN1 gene, which is critical for the survival of motor neurons.
The administration of ITVISMA positions Abu Dhabi as a leader in genomics and precision medicine. It strengthens the UAE’s role in providing advanced care for rare diseases and showcases rapid regulatory and clinical capabilities. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health, emphasized that this achievement underscores the emirate's commitment to world-class healthcare delivery.
The exact cost of ITVISMA has not yet been disclosed. However, it is anticipated to be similar to Zolgensma, another SMA gene therapy priced around $2 million per dose. This raises discussions on:
Gene therapy involves the insertion, alteration, or replacement of genes to treat diseases. It is increasingly utilized for:
Gene therapies are viewed as a frontier area in modern medicine due to their potential for curative outcomes with a single intervention.
Q1. What is ITVISMA gene therapy?
Answer: ITVISMA is a next-generation gene therapy developed by Novartis that targets the genetic cause of spinal muscular atrophy, allowing for the production of essential proteins for motor neuron survival.
Q2. How does spinal muscular atrophy (SMA) affect patients?
Answer: SMA leads to progressive muscle weakness, loss of motor function, and can cause severe complications affecting breathing and swallowing, ultimately posing life-threatening risks.
Q3. What is the significance of Abu Dhabi administering ITVISMA?
Answer: This event positions Abu Dhabi as a leader in genomics and precision medicine, highlighting its rapid regulatory capabilities and commitment to advanced healthcare solutions.
Q4. What are the potential costs associated with ITVISMA?
Answer: While the exact cost of ITVISMA is not disclosed, it is expected to be comparable to Zolgensma, which is priced at approximately $2 million per dose, raising concerns about affordability.
Q5. How is gene therapy changing modern medicine?
Answer: Gene therapy is revolutionizing treatment by offering potential cures for rare genetic disorders and certain cancers, with the ability to provide lasting effects from a single treatment.
Question 1: What is the newly approved gene therapy for spinal muscular atrophy in Abu Dhabi?
A) Zolgensma
B) ITVISMA
C) Spinraza
D) Luxturna
Correct Answer: B
Question 2: Which organization developed ITVISMA gene therapy?
A) Pfizer
B) Novartis
C) Roche
D) Merck
Correct Answer: B
Question 3: What genetic mutation is responsible for spinal muscular atrophy?
A) SMN1
B) BRCA1
C) CFTR
D) TP53
Correct Answer: A
Question 4: How does ITVISMA gene therapy work?
A) It replaces the defective SMN1 gene.
B) It provides pain relief.
C) It enhances muscle strength.
D) It stimulates blood production.
Correct Answer: A
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