A 2-year-old boy is on the road to recovery from the rare muscle-wasting disease spinal muscular atrophy (SMA) after doctors in Dubai gave him a $2.1 million gene therapy injection.
Rasheed's mother said he started showing symptoms of the disease, which is often fatal, when he was less than a year old.
She said she saw Rasheed had issues with his legs. Despite the reassurances of several doctors, he could not crawl, sit, or control his body at the age of 11 months.
The family, who are Egyptian and live in Dubai, took Rasheed to Al Jalila Children’s Specialty Hospital almost 12 months ago, before his first birthday. Medics there diagnosed SMA, a condition that can cause muscle weakness and can affect a child’s ability to develop.
Doctors administered a Zolgensma injection, a prescription gene therapy that is the most expensive life-saving drug in the world, costing about $2.1m.
When Rasheed's clinical team met this month for one-year post-therapy assessment, they found he was demonstrating improved upper limb movement.
“We are pleased with Rasheed’s progress,” said Dr Haitham El Bashir, paediatric neuro-rehabilitation consultant at the hospital.
“He can now move his shoulders against gravity while in a sitting position, he can transit from lying to sitting with minimal assistance from his hips, he can kneel for at least five minutes, and he has started to shuffle his way around on his bottom.”
Rasheed's mother said doctors have told her that improvement levels vary from one child to another, so she needs to patiently monitor her child.
“He has since regained the ability to raise and move his arms. Signs of improvement start from the top of the body to the bottom, from the arms to the waist and eventually the legs,” she said.
August is SMA Awareness Month and Dr El Bashir urged parents to look out for symptoms in their babies such as floppiness, problems with breathing and swallowing, and continual signs of weakness.
“In our experience, the sooner you identify the condition and start the treatment, the better outcome you can achieve,” he said.
“Some children can lose some skills they have gained because they become weaker with time and there is a huge risk because the rapidity of muscle deterioration is such that it can affect breathing and swallowing, which can threaten the child’s life.
“Early identification and treatment are beyond doubt significant to the child’s well-being because a patient treated at two weeks of age is going to do better than one treated at the age of 2.
“That is why it is especially important to have newborn screening. SMA is easy to diagnose through a simple 24-hour genetic test.”
Rasheed’s mother has also urged parents to watch their children from birth for signs of SMA and to seek help immediately if they have concerns.
What are SMA and gene therapy?
Spinal muscular atrophy affects one in 10,000 children and can limit the ability to crawl, walk, sit up, and control head movements.
Children as young as two months can display signs of the most common type of SMA and account for 60 per cent of SMA patients.
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 — 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.
Type 1 is the most severe and many children with it do not live past the age of 2.
Children with Type 2 may sit without support, but cannot walk on their own.
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. They usually have a normal life expectancy.
Type 4 is very rare. It starts in young adulthood and results in mild motor impairment.
Treatment includes the spinal injection that Rasheed received. It is a one-time infusion that transfers the missing survival motor neurone gene directly into body cells through a virus vector — tools commonly used by molecular biologists to deliver genetic material into cells.
In 2019, the Food and Drug Administration in the US approved Zolgensma to treat SMA in children who are younger than 2 years old. It is the first gene therapy to receive FDA approval for the treatment of SMA.
Other recommended treatments include a syrup given to the child for the rest of their life and Spinraza injections, with four injections to be given in the first two months of treatment and then every four months for the rest of the patient’s life.
Spinraza is injected into the fluid surrounding the spinal cord through a needle inserted into the lower back.
The price of Spinraza is $750,000 for the first year and then $350,000 a year after that, giving a total cost of about $4m every decade, drugs.com said.
In the two years since Al Jalila Children’s launched its dedicated gene therapy, the hospital has provided the Zolgensma treatment to 41 patients from 13 countries from across Europe and the Middle East, with the largest number of patients arriving from Turkey.
“It has made a huge difference to the young SMA patients and their families who previously had to travel to the USA for treatment,” Dr El Bashir said.
“In the SMA case, we inject the AAV-9 virus that carries a normal DNA into the cells through an infusion which helps in producing the normal SMN protein needed for the spinal cord neurons to survive.
“Children with SMA have a deficiency of SMN protein, which causes nerves that control muscles — the motor neurons — to die.
"Eventually, their muscles become weak and waste away, with eventual loss of movement and difficulty in breathing and feeding."
The hospital also offers a screening programme study into the epidemiology of SMA in Emirati newborns. It is being undertaken at Genomics Centre, Dubai Health Authority, and private hospitals, which are examining 6,500 newborns across 10 public and private maternity hospitals in the UAE.
The study aims to identify the number of SMA patients who live in the country and project how many there are likely to be in the future, to aid government planning for treatment offerings.
Paul Pogba visits Al Jalila Children’s Specialty Hospital - in pictures
The biog
Favourite hobby: taking his rescue dog, Sally, for long walks.
Favourite book: anything by Stephen King, although he said the films rarely match the quality of the books
Favourite film: The Shawshank Redemption stands out as his favourite movie, a classic King novella
Favourite music: “I have a wide and varied music taste, so it would be unfair to pick a single song from blues to rock as a favourite"
Ultra processed foods
- Carbonated drinks, sweet or savoury packaged snacks, confectionery, mass-produced packaged breads and buns
- margarines and spreads; cookies, biscuits, pastries, cakes, and cake mixes, breakfast cereals, cereal and energy bars;
- energy drinks, milk drinks, fruit yoghurts and fruit drinks, cocoa drinks, meat and chicken extracts and instant sauces
- infant formulas and follow-on milks, health and slimming products such as powdered or fortified meal and dish substitutes,
- many ready-to-heat products including pre-prepared pies and pasta and pizza dishes, poultry and fish nuggets and sticks, sausages, burgers, hot dogs, and other reconstituted meat products, powdered and packaged instant soups, noodles and desserts.
ATP RANKINGS (NOVEMBER 4)
1. Rafael Nadal (ESP) 9,585 pts ( 1)
2. Novak Djokovic (SRB) 8,945 (-1)
3. Roger Federer (SUI) 6,190
4. Daniil Medvedev (RUS) 5,705
5. Dominic Thiem (AUT) 5,025
6. Stefanos Tsitsipas (GRE) 4,000 ( 1)
7. Alexander Zverev (GER) 2,945 (-1)
8. Matteo Berrettini (ITA) 2,670 ( 1)
9. Roberto Bautista (ESP) 2,540 ( 1)
10. Gaël Monfils (FRA) 2,530 ( 3)
11. David Goffin (BEL) 2,335 ( 3)
12. Fabio Fognini (ITA) 2,290
13. Kei Nishikori (JPN) 2,180 (-2)
14. Diego Schwartzman (ARG) 2,125 ( 1)
15. Denis Shapovalov (CAN) 2,050 ( 13)
16. Stan Wawrinka (SUI) 2,000
17. Karen Khachanov (RUS) 1,840 (-9)
18. Alex De Minaur (AUS) 1,775
19. John Isner (USA) 1,770 (-2)
20. Grigor Dimitrov (BUL) 1,747 ( 7)
The specs: 2018 Nissan Patrol Nismo
Price: base / as tested: Dh382,000
Engine: 5.6-litre V8
Gearbox: Seven-speed automatic
Power: 428hp @ 5,800rpm
Torque: 560Nm @ 3,600rpm
Fuel economy, combined: 12.7L / 100km
UAE currency: the story behind the money in your pockets
HWJN
%3Cp%3EDirector%3A%20Yasir%20Alyasiri%3C%2Fp%3E%0A%3Cp%3EStarring%3A%20Baraa%20Alem%2C%20Nour%20Alkhadra%2C%20Alanoud%20Saud%3C%2Fp%3E%0A%3Cp%3ERating%3A%203%2F5%3C%2Fp%3E%0A%3Cp%3E%3C%2Fp%3E%0A
SQUAD
Ali Khaseif, Fahad Al Dhanhani, Adel Al Hosani, Mohammed Al Shamsi, Bandar Al Ahbabi, Mohammed Barghash, Salem Rashid, Khalifa Al Hammadi, Shaheen Abdulrahman, Hassan Al Mahrami, Walid Abbas, Mahmoud Khamis, Yousef Jaber, Saeed Ahmed, Majed Sorour, Majed Hassan, Ali Salmeen, Abdullah Ramadan, Khalil Al Hammadi, Fabio De Lima, Khalfan Mubarak, Tahnoun Al Zaabi, Ali Saleh, Caio Canedo, Muhammed Jumah, Ali Mabkhout, Sebastian Tagliabue, Zayed Al Ameri
How to protect yourself when air quality drops
Install an air filter in your home.
Close your windows and turn on the AC.
Shower or bath after being outside.
Wear a face mask.
Stay indoors when conditions are particularly poor.
If driving, turn your engine off when stationary.
More coverage from the Future Forum
How much do leading UAE’s UK curriculum schools charge for Year 6?
- Nord Anglia International School (Dubai) – Dh85,032
- Kings School Al Barsha (Dubai) – Dh71,905
- Brighton College Abu Dhabi - Dh68,560
- Jumeirah English Speaking School (Dubai) – Dh59,728
- Gems Wellington International School – Dubai Branch – Dh58,488
- The British School Al Khubairat (Abu Dhabi) - Dh54,170
- Dubai English Speaking School – Dh51,269
*Annual tuition fees covering the 2024/2025 academic year
UFC%20FIGHT%20NIGHT%3A%20SAUDI%20ARABIA%20RESULTS
%3Cp%3E%0D%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EMain%20card%3Cbr%3EMiddleweight%3A%3C%2Fstrong%3E%0D%3Cbr%3ERobert%20Whittaker%20defeated%20Ikram%20Aliskerov%20via%20knockout%20(Round%201)%0D%3Cbr%3E%3Cstrong%3EHeavyweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EAlexander%20Volkov%20def%20Sergei%20Pavlovich%20via%20unanimous%20decision%0D%3Cbr%3E%3Cstrong%3EMiddleweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EKelvin%20Gastelum%20def%20Daniel%20Rodriguez%20via%20unanimous%20decision%0D%3Cbr%3E%3Cstrong%3EMiddleweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EShara%20Magomedov%20def%20Antonio%20Trocoli%20via%20knockout%20(Round%203)%0D%3Cbr%3E%3Cstrong%3ELight%20heavyweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EVolkan%20Oezdemir%20def%20Johnny%20Walker%20via%20knockout%20(Round%201)%0D%3Cbr%3E%3Cstrong%3EPreliminary%20Card%0D%3Cbr%3ELightweight%3A%3C%2Fstrong%3E%0D%3Cbr%3ENasrat%20Haqparast%20def%20Jared%20Gordon%20via%20split%20decision%0D%3Cbr%3E%3Cstrong%3EFeatherweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EFelipe%20Lima%20def%20Muhammad%20Naimov%20via%20submission%20(Round%203)%0D%3Cbr%3E%3Cstrong%3EWelterweight%3A%3C%2Fstrong%3E%0D%3Cbr%3ERinat%20Fakhretdinov%20defeats%20Nicolas%20Dalby%20via%20split%20decision%0D%3Cbr%3E%3Cstrong%3EBantamweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EMuin%20Gafurov%20def%20Kang%20Kyung-ho%20via%20unanimous%20decision%0D%3Cbr%3E%3Cstrong%3ELight%20heavyweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EMagomed%20Gadzhiyasulov%20def%20Brendson%20Ribeiro%20via%20majority%20decision%0D%3Cbr%3E%3Cstrong%3EBantamweight%3A%3C%2Fstrong%3E%0D%3Cbr%3EChang%20Ho%20Lee%20def%20Xiao%20Long%20via%20split%20decision%3C%2Fp%3E%0A
The specs
Engine: 4.0-litre V8 twin-turbocharged and three electric motors
Power: Combined output 920hp
Torque: 730Nm at 4,000-7,000rpm
Transmission: 8-speed dual-clutch automatic
Fuel consumption: 11.2L/100km
On sale: Now, deliveries expected later in 2025
Price: expected to start at Dh1,432,000
Which products are to be taxed?
To be taxed:
Flavoured water, long-life fruit juice concentrates, pre-packaged sweetened coffee drinks fall under the ‘sweetened drink’ category
Not taxed
Freshly squeezed fruit juices, ground coffee beans, tea leaves and pre-prepared flavoured milkshakes do not come under the ‘sweetened drink’ band.
Products excluded from the ‘sweetened drink’ category would contain at least 75 per cent milk in a ready-to-drink form or as a milk substitute, baby formula, follow-up formula or baby food, beverages consumed for medicinal use and special dietary needs determined as per GCC Standardisation Organisation rules
The specs
Engine: 6.2-litre V8
Transmission: ten-speed
Power: 420bhp
Torque: 624Nm
Price: Dh325,125
On sale: Now
UAE%20SQUAD
%3Cp%3EMuhammad%20Waseem%20(captain)%2C%20Aayan%20Khan%2C%20Aryan%20Lakra%2C%20Ashwanth%20Valthapa%2C%20Asif%20Khan%2C%20Aryansh%20Sharma%2C%20CP%20Rizwaan%2C%20Hazrat%20Billal%2C%20Junaid%20Siddique%2C%20Karthik%20Meiyappan%2C%20Rohan%20Mustafa%2C%20Vriitya%20Aravind%2C%20Zahoor%20Khan%20and%20Zawar%20Farid.%3C%2Fp%3E%0A
All Black 39-12 British & Irish Lions
RESULTS
5pm: Handicap (TB) Dh100,000, 2,400m
Winner: Recordman, Richard Mullen (jockey), Satish Seemar (trainer)
5.30pm: Wathba Stallions Cup Handicap (PA) Dh 70,000, 2,200m
Winner: AF Taraha, Tadhg O’Shea, Ernst Oertel
6pm: Abu Dhabi Fillies Classic Prestige (PA) Dh110,000, 1,400m
Winner: Dhafra, Fabrice Veron, Eric Lemartinel
6.30pm: Abu Dhabi Colts Classic Prestige (PA) Dh110,000, 1,400m
Winner: Maqam, Fabrice Veron, Eric Lemartinel
7pm: Handicap (PA) Dh85,000, 1,600m
Winner: AF Momtaz, Fernando Jara, Musabah Al Muhairi
7.30pm: Maiden (PA) Dh80,000, 1,600m
Winner: Optimizm, Patrick Cosgrave, Abdallah Al Hammadi
Killing of Qassem Suleimani
Fines for littering
In Dubai:
Dh200 for littering or spitting in the Dubai Metro
Dh500 for throwing cigarette butts or chewing gum on the floor, or littering from a vehicle.
Dh1,000 for littering on a beach, spitting in public places, throwing a cigarette butt from a vehicle
In Sharjah and other emirates
Dh500 for littering - including cigarette butts and chewing gum - in public places and beaches in Sharjah
Dh2,000 for littering in Sharjah deserts
Dh500 for littering from a vehicle in Ras Al Khaimah
Dh1,000 for littering from a car in Abu Dhabi
Dh1,000 to Dh100,000 for dumping waste in residential or public areas in Al Ain
Dh10,000 for littering at Ajman's beaches
Mental%20health%20support%20in%20the%20UAE
%3Cp%3E%E2%97%8F%20Estijaba%20helpline%3A%208001717%3Cbr%3E%E2%97%8F%20UAE%20Ministry%20of%20Health%20and%20Prevention%20hotline%3A%20045192519%3Cbr%3E%E2%97%8F%20UAE%20Mental%20health%20support%20line%3A%20800%204673%20(Hope)%3Cbr%3EMore%20information%20at%20hope.hw.gov.ae%3C%2Fp%3E%0A
STAGE%201%20RESULTS
%3Cp%3E1)%20Tim%20Merlier%20(Soudal-Quick-Step)%2C%203h%2017%E2%80%99%2035%E2%80%9D%3Cbr%3E2)%20Caleb%20Ewan%20(Lotto%20Dstny)%20same%20time%3Cbr%3E3)%20Mark%20Cavendish%20(Astana%20Qazaqstan%20Team)%20same%20time%3Cbr%3E%3Cstrong%3EGeneral%20Classification%3A%3C%2Fstrong%3E%3Cbr%3E1)%20Tim%20Merlier%20(Soudal%20Quick-Step)%203%3A17%3A25%3Cbr%3E2%20-%20Caleb%20Ewan%20(Lotto%20Dstny)%20%2B4%22%3Cbr%3E3%20-%20Luke%20Plapp%20(Ineos%20Grenadiers)%20%2B5%22%3C%2Fp%3E%0A
<html><head><meta http-equiv="Content-Type" content="text/html" charset="UTF-8" /></head><body><!--PSTYLE=* Labels%3aFH Label 18 Sport--><p>Beach soccer</p><!--PSTYLE=BY Byline--><p>Amith Passela</p><p /></body></html>
DUBAI CARNIVAL RESULTS
6.30pm Handicap US$135,000 (Turf) 2,410m
Winner Dubai Future, Harry Bentley (jockey), Saeed bin Suroor (trainer).
7.05pm UAE 1000 Guineas Listed $250,000 (Dirt) 1,600m
Winner Dubai Love, Patrick Cosgrave, Saeed bin Suroor.
7.40pm Dubai Dash Listed $175,000 (T) 1,000m
Winner: Equilateral, James Doyle, Charles Hills.
8.15pm Al Bastakiya Trial Conditions $100,000 (D) 1.900m
Winner Laser Show, Kevin Stott, Saeed bin Suroor.
8.50pm Al Fahidi Fort Group Two $250,000 (T) 1,400m
Winner Glorious Journey, James Doyle, Charlie Appleby.
9.25pm Handicap $135,000 (D) 2,000m
Winner George Villiers, Tadhg O’Shea, Satish Seemar.
Planes grounded by coronavirus
British Airways: Cancels all direct flights to and from mainland China
Hong Kong-based Cathay Pacific: Cutting capacity to/from mainland China by 50 per cent from Jan. 30
Chicago-based United Airlines: Reducing flights to Beijing, Shanghai, and Hong Kong
Ai Seoul: Suspended all flights to China
Finnair: Suspending flights to Nanjing and Beijing Daxing until the end of March
Indonesia's Lion Air: Suspending all flights to China from February
South Korea's Asiana Airlines, Jeju Air and Jin Air: Suspend all flights
US households add $601bn of debt in 2019
American households borrowed another $601 billion (Dh2.2bn) in 2019, the largest yearly gain since 2007, just before the global financial crisis, according to February data from the New York Federal Reserve Bank.
Fuelled by rising mortgage debt as homebuyers continued to take advantage of low interest rates, the increase last year brought total household debt to a record high, surpassing the previous peak reached in 2008 just before the market crash, according to the report.
Following the 22nd straight quarter of growth, American household debt swelled to $14.15 trillion by the end of 2019, the New York Fed said in its quarterly report.
In the final three months of the year, new home loans jumped to their highest volume since the fourth quarter of 2005, while credit cards and auto loans also added to the increase.
The bad debt load is taking its toll on some households, and the New York Fed warned that more and more credit card borrowers — particularly young people — were falling behind on their payments.
"Younger borrowers, who are disproportionately likely to have credit cards and student loans as their primary form of debt, struggle more than others with on-time repayment," New York Fed researchers said.
The Bio
Name: Lynn Davison
Profession: History teacher at Al Yasmina Academy, Abu Dhabi
Children: She has one son, Casey, 28
Hometown: Pontefract, West Yorkshire in the UK
Favourite book: The Alchemist by Paulo Coelho
Favourite Author: CJ Sansom
Favourite holiday destination: Bali
Favourite food: A Sunday roast
TRAINING FOR TOKYO
A typical week's training for Sebastian, who is competing at the ITU Abu Dhabi World Triathlon on March 8-9:
- Four swim sessions (14km)
- Three bike sessions (200km)
- Four run sessions (45km)
- Two strength and conditioning session (two hours)
- One session therapy session at DISC Dubai
- Two-three hours of stretching and self-maintenance of the body
ITU Abu Dhabi World Triathlon
For more information go to www.abudhabi.triathlon.org.
