Spinal muscular atrophy (SMA) is a debilitating condition that affects around one in every 10,000 children.
Zolgensma, the most effective treatment, is also the world’s most expensive drug.
A single one-time infusion of the drug costs Dh8 million.
But why does it cost so much?
The National explains.
What is SMA?
SMA is a hereditary disease caused by a missing or faulty gene that the body requires to make a protein essential for motor neuron cell survival.
Without sufficient levels of the protein, the motor neurons - which are nerve cells in the brain stem and spinal cord that control activities such as speaking, walking, breathing, and swallowing - die, leading to muscle weakness and atrophy.
There are four types of the condition.
These therapies are extraordinarily expensive as it is first of its kind gene modifying therapy, approved for rare neuromuscular disorders
Dr Sushil Garg,
NMC Royal Hospital
Type 1 is the most severe and many children with it do not live past the age of two. In July, The National reported on the case of three-month-old Malak Al Alami, who received Zolgensma after winning a global “lottery” run by the medicine’s manufacturer, Novartis. It is allocating up to 100 doses at no charge this year and has appointed a third party to select recipients every two weeks.
Children with Type 2 may sit without support, but cannot walk on their own. Symptoms typically appear between six and 18 months. Depending on the severity of their symptoms, children with this type may live a normal life span.
Type 3 is a milder form of the disease and resembles muscular dystrophy. Children are usually able to walk with difficulty and some eventually need a wheelchair. Symptoms usually appear around 18 months or in early childhood. They usually have a normal life expectancy.
Type 4 is very rare. It starts in young adulthood and results in mild motor impairment.
How does the treatment work and why is it so expensive?
Zolgensma is the only therapy designed to directly address the genetic root cause of the condition. It does this by replacing the function of the missing or nonworking gene, which is critical in making a protein essential to motor neuron cell survival.
It works by using a harmless, genetically engineered virus that travels around the body to help restore some of the protein missing as a result of the condition.
It is extremely effective, but hugely expensive because its market is very small and it required a lot of research to develop it, Dr Sushil Garg, a consultant neurologist at NMC Royal Hospital in Dubai, told The National.
“These therapies are extraordinarily expensive as it is first of its kind gene modifying therapy, approved for rare neuromuscular disorders,” said Dr Garg.
“Any medicine which is for gene modifying or work on [the] immune system has to undergo a lot of research and trials and approval processes, hence is pretty expensive.”
However, it currently is only used in children under two, but that may change in time.
New trial
Novartis launched a trial earlier this year to evaluate the safety and efficacy of the drug in children with SMA weighing between 8.5kg and 21kg.
The results will supplement emerging real-world evidence and use of the therapy in the European Union and Canada, where regulators have approved the drug for use in babies and young children up to 21kg.
Abu Dhabi residents Rahul Pathak, 37 and his wife Astha Muthoo, 35, hope it will help their six-year-old daughter, Arya Pathak, who suffers from the condition. The National covered her plight earlier this week.
The disease has forced her to spend weeks at a time in intensive care. Even a common cold can take a heavy toll on her weakened body.
Her parents cannot legally fundraise to help cover the cost of the drug, but donors can pay money directly to hospitals to help with medical bills.
Libya's Gold
UN Panel of Experts found regime secretly sold a fifth of the country's gold reserves.
The panel’s 2017 report followed a trail to West Africa where large sums of cash and gold were hidden by Abdullah Al Senussi, Qaddafi’s former intelligence chief, in 2011.
Cases filled with cash that was said to amount to $560m in 100 dollar notes, that was kept by a group of Libyans in Ouagadougou, Burkina Faso.
A second stash was said to have been held in Accra, Ghana, inside boxes at the local offices of an international human rights organisation based in France.
Company%20profile
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Sarfira
Director: Sudha Kongara Prasad
Starring: Akshay Kumar, Radhika Madan, Paresh Rawal
Rating: 2/5
The biog
From: Upper Egypt
Age: 78
Family: a daughter in Egypt; a son in Dubai and his wife, Nabila
Favourite Abu Dhabi activity: walking near to Emirates Palace
Favourite building in Abu Dhabi: Emirates Palace
BMW M5 specs
Engine: 4.4-litre twin-turbo V-8 petrol enging with additional electric motor
Power: 727hp
Torque: 1,000Nm
Transmission: 8-speed auto
Fuel consumption: 10.6L/100km
On sale: Now
Price: From Dh650,000
The specs: 2018 Volkswagen Teramont
Price, base / as tested Dh137,000 / Dh189,950
Engine 3.6-litre V6
Gearbox Eight-speed automatic
Power 280hp @ 6,200rpm
Torque 360Nm @ 2,750rpm
Fuel economy, combined 11.7L / 100km
HIJRA
Starring: Lamar Faden, Khairiah Nathmy, Nawaf Al-Dhufairy
Director: Shahad Ameen
Rating: 3/5
Sly%20Cooper%20and%20the%20Thievius%20Raccoonus
%3Cp%3E%3Cstrong%3EDeveloper%3A%3C%2Fstrong%3E%20Sucker%20Punch%20Productions%3Cbr%3E%3Cstrong%3EPublisher%3A%3C%2Fstrong%3E%20Sony%20Computer%20Entertainment%3Cbr%3E%3Cstrong%3EConsole%3A%3C%2Fstrong%3E%20PlayStation%202%20to%205%3Cbr%3E%3Cstrong%3ERating%3A%3C%2Fstrong%3E%205%2F5%3C%2Fp%3E%0A
Results:
5pm: Conditions (PA) Dh80,000 1,400m | Winner: AF Tahoonah, Richard Mullen (jockey), Ernst Oertel (trainer)
5.30pm: Handicap (TB) Dh90,000 1,400m | Winner: Ajwad, Gerald Avranche, Rashed Bouresly
6pm: Maiden (PA) Dh80,000 1,600m | Winner: RB Lam Tara, Fabrice Veron, Eric Lemartinel
6.30pm: Handicap (PA) Dh80,000 1,600m | Winner: Duc De Faust, Szczepan Mazur, Younis Al Kalbani
7pm: Wathba Stallions Cup (PA) Dh70,000 2,200m | Winner: Shareef KB, Fabrice Veron, Ernst Oertel
7.30pm: Handicap (PA) Dh90,000 1,500m | Winner: Bainoona, Pat Cosgrave, Eric Lemartinel
The biog
Name: Fareed Lafta
Age: 40
From: Baghdad, Iraq
Mission: Promote world peace
Favourite poet: Al Mutanabbi
Role models: His parents
Engine: 3.5-litre V6
Transmission: eight-speed automatic
Power: 290hp
Torque: 340Nm
Price: Dh155,800
On sale: now
Match info
Huddersfield Town 0
Chelsea 3
Kante (34'), Jorginho (45' pen), Pedro (80')
UAE currency: the story behind the money in your pockets
'Champions'
Director: Manuel Calvo
Stars: Yassir Al Saggaf and Fatima Al Banawi
Rating: 2/5
Teri%20Baaton%20Mein%20Aisa%20Uljha%20Jiya
%3Cp%3E%3Cstrong%3EDirectors%3A%3C%2Fstrong%3E%20Amit%20Joshi%20and%20Aradhana%20Sah%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3ECast%3A%3C%2Fstrong%3E%20Shahid%20Kapoor%2C%20Kriti%20Sanon%2C%20Dharmendra%2C%20Dimple%20Kapadia%2C%20Rakesh%20Bedi%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3ERating%3A%3C%2Fstrong%3E%204%2F5%3C%2Fp%3E%0A
The Bio
Amal likes watching Japanese animation movies and Manga - her favourite is The Ancient Magus Bride
She is the eldest of 11 children, and has four brothers and six sisters.
Her dream is to meet with all of her friends online from around the world who supported her work throughout the years
Her favourite meal is pizza and stuffed vine leaves
She ams to improve her English and learn Japanese, which many animated programmes originate in
Skoda Superb Specs
Engine: 2-litre TSI petrol
Power: 190hp
Torque: 320Nm
Price: From Dh147,000
Available: Now
Ten tax points to be aware of in 2026
1. Domestic VAT refund amendments: request your refund within five years
If a business does not apply for the refund on time, they lose their credit.
2. E-invoicing in the UAE
Businesses should continue preparing for the implementation of e-invoicing in the UAE, with 2026 a preparation and transition period ahead of phased mandatory adoption.
3. More tax audits
Tax authorities are increasingly using data already available across multiple filings to identify audit risks.
4. More beneficial VAT and excise tax penalty regime
Tax disputes are expected to become more frequent and more structured, with clearer administrative objection and appeal processes. The UAE has adopted a new penalty regime for VAT and excise disputes, which now mirrors the penalty regime for corporate tax.
5. Greater emphasis on statutory audit
There is a greater need for the accuracy of financial statements. The International Financial Reporting Standards standards need to be strictly adhered to and, as a result, the quality of the audits will need to increase.
6. Further transfer pricing enforcement
Transfer pricing enforcement, which refers to the practice of establishing prices for internal transactions between related entities, is expected to broaden in scope. The UAE will shortly open the possibility to negotiate advance pricing agreements, or essentially rulings for transfer pricing purposes.
7. Limited time periods for audits
Recent amendments also introduce a default five-year limitation period for tax audits and assessments, subject to specific statutory exceptions. While the standard audit and assessment period is five years, this may be extended to up to 15 years in cases involving fraud or tax evasion.
8. Pillar 2 implementation
Many multinational groups will begin to feel the practical effect of the Domestic Minimum Top-Up Tax (DMTT), the UAE's implementation of the OECD’s global minimum tax under Pillar 2. While the rules apply for financial years starting on or after January 1, 2025, it is 2026 that marks the transition to an operational phase.
9. Reduced compliance obligations for imported goods and services
Businesses that apply the reverse-charge mechanism for VAT purposes in the UAE may benefit from reduced compliance obligations.
10. Substance and CbC reporting focus
Tax authorities are expected to continue strengthening the enforcement of economic substance and Country-by-Country (CbC) reporting frameworks. In the UAE, these regimes are increasingly being used as risk-assessment tools, providing tax authorities with a comprehensive view of multinational groups’ global footprints and enabling them to assess whether profits are aligned with real economic activity.
Contributed by Thomas Vanhee and Hend Rashwan, Aurifer
Dhadak 2
Director: Shazia Iqbal
Starring: Siddhant Chaturvedi, Triptii Dimri
Rating: 1/5
Sri Lanka v England
First Test, at Galle
England won by 211
Second Test, at Kandy
England won by 57 runs
Third Test, at Colombo
From Nov 23-27
What vitamins do we know are beneficial for living in the UAE
Vitamin D: Highly relevant in the UAE due to limited sun exposure; supports bone health, immunity and mood.
Vitamin B12: Important for nerve health and energy production, especially for vegetarians, vegans and individuals with absorption issues.
Iron: Useful only when deficiency or anaemia is confirmed; helps reduce fatigue and support immunity.
Omega-3 (EPA/DHA): Supports heart health and reduces inflammation, especially for those who consume little fish.
Mercer, the investment consulting arm of US services company Marsh & McLennan, expects its wealth division to at least double its assets under management (AUM) in the Middle East as wealth in the region continues to grow despite economic headwinds, a company official said.
Mercer Wealth, which globally has $160 billion in AUM, plans to boost its AUM in the region to $2-$3bn in the next 2-3 years from the present $1bn, said Yasir AbuShaban, a Dubai-based principal with Mercer Wealth.
“Within the next two to three years, we are looking at reaching $2 to $3 billion as a conservative estimate and we do see an opportunity to do so,” said Mr AbuShaban.
Mercer does not directly make investments, but allocates clients’ money they have discretion to, to professional asset managers. They also provide advice to clients.
“We have buying power. We can negotiate on their (client’s) behalf with asset managers to provide them lower fees than they otherwise would have to get on their own,” he added.
Mercer Wealth’s clients include sovereign wealth funds, family offices, and insurance companies among others.
From its office in Dubai, Mercer also looks after Africa, India and Turkey, where they also see opportunity for growth.
Wealth creation in Middle East and Africa (MEA) grew 8.5 per cent to $8.1 trillion last year from $7.5tn in 2015, higher than last year’s global average of 6 per cent and the second-highest growth in a region after Asia-Pacific which grew 9.9 per cent, according to consultancy Boston Consulting Group (BCG). In the region, where wealth grew just 1.9 per cent in 2015 compared with 2014, a pickup in oil prices has helped in wealth generation.
BCG is forecasting MEA wealth will rise to $12tn by 2021, growing at an annual average of 8 per cent.
Drivers of wealth generation in the region will be split evenly between new wealth creation and growth of performance of existing assets, according to BCG.
Another general trend in the region is clients’ looking for a comprehensive approach to investing, according to Mr AbuShaban.
“Institutional investors or some of the families are seeing a slowdown in the available capital they have to invest and in that sense they are looking at optimizing the way they manage their portfolios and making sure they are not investing haphazardly and different parts of their investment are working together,” said Mr AbuShaban.
Some clients also have a higher appetite for risk, given the low interest-rate environment that does not provide enough yield for some institutional investors. These clients are keen to invest in illiquid assets, such as private equity and infrastructure.
“What we have seen is a desire for higher returns in what has been a low-return environment specifically in various fixed income or bonds,” he said.
“In this environment, we have seen a de facto increase in the risk that clients are taking in things like illiquid investments, private equity investments, infrastructure and private debt, those kind of investments were higher illiquidity results in incrementally higher returns.”
The Abu Dhabi Investment Authority, one of the largest sovereign wealth funds, said in its 2016 report that has gradually increased its exposure in direct private equity and private credit transactions, mainly in Asian markets and especially in China and India. The authority’s private equity department focused on structured equities owing to “their defensive characteristics.”
CHATGPT%20ENTERPRISE%20FEATURES
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Getting%20there%20
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Company%20profile
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More on animal trafficking
Living in...
This article is part of a guide on where to live in the UAE. Our reporters will profile some of the country’s most desirable districts, provide an estimate of rental prices and introduce you to some of the residents who call each area home.
Springsteen: Deliver Me from Nowhere
Director: Scott Cooper
Starring: Jeremy Allen White, Odessa Young, Jeremy Strong
Rating: 4/5
More from Neighbourhood Watch
Key findings of Jenkins report
- Founder of the Muslim Brotherhood, Hassan al Banna, "accepted the political utility of violence"
- Views of key Muslim Brotherhood ideologue, Sayyid Qutb, have “consistently been understood” as permitting “the use of extreme violence in the pursuit of the perfect Islamic society” and “never been institutionally disowned” by the movement.
- Muslim Brotherhood at all levels has repeatedly defended Hamas attacks against Israel, including the use of suicide bombers and the killing of civilians.
- Laying out the report in the House of Commons, David Cameron told MPs: "The main findings of the review support the conclusion that membership of, association with, or influence by the Muslim Brotherhood should be considered as a possible indicator of extremism."
Graduated from the American University of Sharjah
She is the eldest of three brothers and two sisters
Has helped solve 15 cases of electric shocks
Enjoys travelling, reading and horse riding