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.
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.
Killing of Qassem Suleimani
National Editorial: Suleimani has been killed, now we must de-escalate
Mina Al Oraibi: Air strike casts a long shadow over the decade ahead
Jack Moore: Why the assassination is such a monumental gamble
Damien McElroy: A CEO tasked with spreading Iran's influence
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Simon Waldman: Cautious Israel keeping a low profile
World Mental Health Day
• Remittance charges will be tackled by blockchain
• UAE's monumental and risky Mars Mission to inspire future generations, says minister
• Could the UAE drive India's economy?
• News has a bright future and the UAE is at the heart of it
• Architecture is over - here's cybertecture
• The National announces Future of News journalism competition
• Round up: Experts share their visions of the world to come
Tomorrow 2021
Plastic tipping points
Milestones on the road to union
1970
October 26: Bahrain withdraws from a proposal to create a federation of nine with the seven Trucial States and Qatar.
December: Ahmed Al Suwaidi visits New York to discuss potential UN membership.
1971
March 1: Alex Douglas Hume, Conservative foreign secretary confirms that Britain will leave the Gulf and “strongly supports” the creation of a Union of Arab Emirates.
July 12: Historic meeting at which Sheikh Zayed and Sheikh Rashid make a binding agreement to create what will become the UAE.
July 18: It is announced that the UAE will be formed from six emirates, with a proposed constitution signed. RAK is not yet part of the agreement.
August 6: The fifth anniversary of Sheikh Zayed becoming Ruler of Abu Dhabi, with official celebrations deferred until later in the year.
August 15: Bahrain becomes independent.
September 3: Qatar becomes independent.
November 23-25: Meeting with Sheikh Zayed and Sheikh Rashid and senior British officials to fix December 2 as date of creation of the UAE.
November 29: At 5.30pm Iranian forces seize the Greater and Lesser Tunbs by force.
November 30: Despite a power sharing agreement, Tehran takes full control of Abu Musa.
November 31: UK officials visit all six participating Emirates to formally end the Trucial States treaties
December 2: 11am, Dubai. New Supreme Council formally elects Sheikh Zayed as President. Treaty of Friendship signed with the UK. 11.30am. Flag raising ceremony at Union House and Al Manhal Palace in Abu Dhabi witnessed by Sheikh Khalifa, then Crown Prince of Abu Dhabi.
December 6: Arab League formally admits the UAE. The first British Ambassador presents his credentials to Sheikh Zayed.
December 9: UAE joins the United Nations.
Read more from Aya Iskandarani
Company%C2%A0profile
Du Football Champions
The fourth season of du Football Champions was launched at Gitex on Wednesday alongside the Middle East’s first sports-tech scouting platform.“du Talents”, which enables aspiring footballers to upload their profiles and highlights reels and communicate directly with coaches, is designed to extend the reach of the programme, which has already attracted more than 21,500 players in its first three years.
RESULT
RS Leipzig 3
Marcel Sabitzer 10', 21'
Emil Forsberg 87'
Tottenham 0
Killing of Qassem Suleimani
National Editorial: Suleimani has been killed, now we must de-escalate
Jack Moore: Why the assassination is such a monumental gamble
Matthew Levitt: Iran retains its ability to launch terror attacks
Damien McElroy: A CEO tasked with spreading Iran's influence
Hussein Ibish: Trump's order on solid constitutional ground
Simon Waldman: Cautious Israel keeping a low profile
Transgender report
MADAME%20WEB
MATCH INFO
Norwich 0
Watford 2 (Deulofeu 2', Gray 52')
Red card: Christian Kabasele (WatforD)
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SPECS
Engine: Two-litre four-cylinder turbo
Power: 235hp
Torque: 350Nm
Transmission: Nine-speed automatic
Price: From Dh167,500 ($45,000)
On sale: Now
Transgender report
Kareem Shaheen on Canada
On Women's Day
Shelina Janmohamed: Why shouldn't a spouse be compensated fairly for housework?
Samar Elmnhrawy: How companies in the Middle East can catch up on gender equality
The National Editorial: Is there much to celebrate on International Women's Day 2021?
Justin Thomas: Challenge the notion that 'men are from Mars, women are from Venus'
Killing of Qassem Suleimani
National Editorial: Suleimani has been killed, now we must de-escalate
Mina Al Oraibi: Air strike casts a long shadow over the decade ahead
Jack Moore: Why the assassination is such a monumental gamble
Matthew Levitt: Iran retains its ability to launch terror attacks
Hussein Ibish: Trump's order on solid constitutional ground
Simon Waldman: Cautious Israel keeping a low profile
Our commentary on Brexit
- Alistair Burt: Despite Brexit, Britain can remain a world power
- Con Coughlin: Choice of the British people will be vindicated
What sanctions would be reimposed?
Under ‘snapback’, measures imposed on Iran by the UN Security Council in six resolutions would be restored, including:
- An arms embargo
- A ban on uranium enrichment and reprocessing
- A ban on launches and other activities with ballistic missiles capable of delivering nuclear weapons, as well as ballistic missile technology transfer and technical assistance
- A targeted global asset freeze and travel ban on Iranian individuals and entities
- Authorisation for countries to inspect Iran Air Cargo and Islamic Republic of Iran Shipping Lines cargoes for banned goods
Plastic tipping point
Jetour T1 specs
Engine: 2-litre turbocharged
Power: 254hp
Torque: 390Nm
Price: From Dh126,000
Available: Now
Previous men's records
- 2:01:39: Eliud Kipchoge (KEN) on 16/9/19 in Berlin
- 2:02:57: Dennis Kimetto (KEN) on 28/09/2014 in Berlin
- 2:03:23: Wilson Kipsang (KEN) on 29/09/2013 in Berlin
- 2:03:38: Patrick Makau (KEN) on 25/09/2011 in Berlin
- 2:03:59: Haile Gebreselassie (ETH) on 28/09/2008 in Berlin
- 2:04:26: Haile Gebreselassie (ETH) on 30/09/2007 in Berlin
- 2:04:55: Paul Tergat (KEN) on 28/09/2003 in Berlin
- 2:05:38: Khalid Khannouchi (USA) 14/04/2002 in London
- 2:05:42: Khalid Khannouchi (USA) 24/10/1999 in Chicago
- 2:06:05: Ronaldo da Costa (BRA) 20/09/1998 in Berlin
• Remittance charges will be tackled by blockchain
• UAE's monumental and risky Mars Mission to inspire future generations, says minister
• Could the UAE drive India's economy?
• News has a bright future and the UAE is at the heart of it
• Architecture is over - here's cybertecture
• The National announces Future of News journalism competition
• Round up: Experts share their visions of the world to come
The burning issue
The internal combustion engine is facing a watershed moment – major manufacturer Volvo is to stop producing petroleum-powered vehicles by 2021 and countries in Europe, including the UK, have vowed to ban their sale before 2040. The National takes a look at the story of one of the most successful technologies of the last 100 years and how it has impacted life in the UAE.
Read part four: an affection for classic cars lives on
Read part three: the age of the electric vehicle begins
Read part one: how cars came to the UAE
Mohammed bin Zayed Majlis
Results
7pm: Wathba Stallions Cup – Handicap (PA) Dh70,000 (Dirt) 1,600m; Winner: RB Kings Bay, Abdul Aziz Al Balushi (jockey), Helal Al Alawi (trainer)
7.30pm: Maiden (PA) Dh 70,000 (D) 1,600m; Winner: AF Ensito, Fernando Jara, Mohamed Daggash
8pm: Maiden (PA) Dh70,000 (D) 1,400m; Winner: AF Sourouh, Tadhg O’Shea, Ernst Oertel
8.30pm: Maiden (PA) Dh70,000 (D) 1,800m; Winner: Baaher, Fabrice Veron, Eric Lemartinel
9pm: Maiden (PA) Dh70,000 (D) 2,000m; Winner: Mootahady, Antonio Fresu, Eric Lemartinel
9.30pm: Handicap (TB) Dh70,000 (D) 2,000m; Winner: Dubai Canal, Tadhg O’Shea, Satish Seemar
10pm: Al Ain Cup – Prestige (PA) Dh100,000 (D) 2,000m; Winner: Harrab, Bernardo Pinheiro, Majed Al Jahouri
Company name: Play:Date
Launched: March 2017 on UAE Mother’s Day
Founder: Shamim Kassibawi
Based: Dubai with operations in the UAE and US
Sector: Tech
Size: 20 employees
Stage of funding: Seed
Investors: Three founders (two silent co-founders) and one venture capital fund
While you're here
Kareem Shaheen: Even a pandemic could not unite today's America
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MATCH INFO
Newcastle United 3
Gayle (23'), Perez (59', 63')
Chelsea 0
More on Quran memorisation:
Plastic tipping points
The Bio
Hometown: Bogota, Colombia
Favourite place to relax in UAE: the desert around Al Mleiha in Sharjah or the eastern mangroves in Abu Dhabi
The one book everyone should read: 100 Years of Solitude by Gabriel Garcia Marquez. It will make your mind fly
Favourite documentary: Chasing Coral by Jeff Orlowski. It's a good reality check about one of the most valued ecosystems for humanity
• Remittance charges will be tackled by blockchain
• UAE's monumental and risky Mars Mission to inspire future generations, says minister
• Could the UAE drive India's economy?
• News has a bright future and the UAE is at the heart of it
• Architecture is over - here's cybertecture
• The National announces Future of News journalism competition
• Round up: Experts share their visions of the world to come
Heavily-sugared soft drinks slip through the tax net
Some popular drinks with high levels of sugar and caffeine have slipped through the fizz drink tax loophole, as they are not carbonated or classed as an energy drink.
Arizona Iced Tea with lemon is one of those beverages, with one 240 millilitre serving offering up 23 grams of sugar - about six teaspoons.
A 680ml can of Arizona Iced Tea costs just Dh6.
Most sports drinks sold in supermarkets were found to contain, on average, five teaspoons of sugar in a 500ml bottle.
While you're here
Samanth Subramanian: Imposing Hindi could undermine India's linguistic diversity
Phil Mercer: Aboriginal musicians are struggling to be heard
Gavin Esler: What we lose when we allow languages to die
Tomorrow 2021
EMERGENCY PHONE NUMBERS
Estijaba – 8001717 – number to call to request coronavirus testing
Ministry of Health and Prevention – 80011111
Dubai Health Authority – 800342 – The number to book a free video or voice consultation with a doctor or connect to a local health centre
Emirates airline – 600555555
Etihad Airways – 600555666
Ambulance – 998
Knowledge and Human Development Authority – 8005432 ext. 4 for Covid-19 queries
Killing of Qassem Suleimani
National Editorial: Suleimani has been killed, now we must de-escalate
Mina Al Oraibi: Air strike casts a long shadow over the decade ahead
Matthew Levitt: Iran retains its ability to launch terror attacks
Damien McElroy: A CEO tasked with spreading Iran's influence
Hussein Ibish: Trump's order on solid constitutional ground
Simon Waldman: Cautious Israel keeping a low profile
• Remittance charges will be tackled by blockchain
• UAE's monumental and risky Mars Mission to inspire future generations, says minister
• Could the UAE drive India's economy?
• News has a bright future and the UAE is at the heart of it
• Architecture is over - here's cybertecture
• The National announces Future of News journalism competition
• Round up: Experts share their visions of the world to come
More on animal trafficking
More from Neighbourhood Watch:
SERIES INFO
Afghanistan v Zimbabwe, Abu Dhabi Sunshine Series
All matches at the Zayed Cricket Stadium, Abu Dhabi
Test series
1st Test: Zimbabwe beat Afghanistan by 10 wickets
2nd Test: Wednesday, 10 March – Sunday, 14 March
Play starts at 9.30am
T20 series
1st T20I: Wednesday, 17 March
2nd T20I: Friday, 19 March
3rd T20I: Saturday, 20 March
TV
Supporters in the UAE can watch the matches on the Rabbithole channel on YouTube
While you're here
On Women's Day
Dr Nawal Al-Hosany: Why more women should be on the frontlines of climate action
Shelina Janmohamed: Why shouldn't a spouse be compensated fairly for housework?
Justin Thomas: Challenge the notion that 'men are from Mars, women are from Venus'
The National Editorial: Is there much to celebrate on International Women's Day 2021?
On Women's Day
Dr Nawal Al-Hosany: Why more women should be on the frontlines of climate action
Shelina Janmohamed: Why shouldn't a spouse be compensated fairly for housework?
Samar Elmnhrawy: How companies in the Middle East can catch up on gender equality
Justin Thomas: Challenge the notion that 'men are from Mars, women are from Venus'
Whiile you're here
Damien McElroy: Anti-science attitudes in America are proving lethal
Editorial: What makes the UAE such a good place to test vaccines?
Editorial: The fight against Covid-19 should be guided by science
Zayed Sustainability Prize
How it works
1) The liquid nanoclay is a mixture of water and clay that aims to convert desert land to fertile ground
2) Instead of water draining straight through the sand, it apparently helps the soil retain water
3) One application is said to last five years
4) The cost of treatment per hectare (2.4 acres) of desert varies from $7,000 to $10,000 per hectare
Key facilities
- Olympic-size swimming pool with a split bulkhead for multi-use configurations, including water polo and 50m/25m training lanes
- Premier League-standard football pitch
- 400m Olympic running track
- NBA-spec basketball court with auditorium
- 600-seat auditorium
- Spaces for historical and cultural exploration
- An elevated football field that doubles as a helipad
- Specialist robotics and science laboratories
- AR and VR-enabled learning centres
- Disruption Lab and Research Centre for developing entrepreneurial skills
Farage on Muslim Brotherhood
Nigel Farage told Reform's annual conference that the party will proscribe the Muslim Brotherhood if he becomes Prime Minister.
"We will stop dangerous organisations with links to terrorism operating in our country," he said. "Quite why we've been so gutless about this – both Labour and Conservative – I don't know.
“All across the Middle East, countries have banned and proscribed the Muslim Brotherhood as a dangerous organisation. We will do the very same.”
It is 10 years since a ground-breaking report into the Muslim Brotherhood by Sir John Jenkins.
Among the former diplomat's findings was an assessment that “the use of extreme violence in the pursuit of the perfect Islamic society” has “never been institutionally disowned” by the movement.
The prime minister at the time, David Cameron, who commissioned the report, said membership or association with the Muslim Brotherhood was a "possible indicator of extremism" but it would not be banned.
COMPANY%20PROFILE
On Women's Day
Dr Nawal Al-Hosany: Why more women should be on the frontlines of climate action
Samar Elmnhrawy: How companies in the Middle East can catch up on gender equality
The National Editorial: Is there much to celebrate on International Women's Day 2021?
Justin Thomas: Challenge the notion that 'men are from Mars, women are from Venus'
Panipat
Director Ashutosh Gowariker
Produced Ashutosh Gowariker, Rohit Shelatkar, Reliance Entertainment
Cast Arjun Kapoor, Sanjay Dutt, Kriti Sanon, Mohnish Behl, Padmini Kolhapure, Zeenat Aman
Rating 3 /5 stars
Mohammed bin Zayed Majlis
Tomorrow 2021
UAE v Ireland
1st ODI, UAE win by 6 wickets
2nd ODI, January 12
3rd ODI, January 14
4th ODI, January 16
KILLING OF QASSEM SULEIMANI
National Editorial: Suleimani has been killed, now we must de-escalate
Mina Al Oraibi: Air strike casts a long shadow over the decade ahead
Jack Moore: Why the assassination is such a monumental gamble
Matthew Levitt: Iran retains its ability to launch terror attacks
Damien McElroy: A CEO tasked with spreading Iran's influence
Hussein Ibish: Trump's order on solid constitutional ground
Simon Waldman: Cautious Israel keeping a low profile
Teams
Pakistan: Sarfraz Ahmed (captain), Mohammad Hafeez, Sahibzada Farhan, Babar Azam, Shoaib Malik, Asif Ali, Shadab Khan, Shaheen Shah Afridi, Usman Khan Shanwari, Hasan Ali, Imad Wasim, Faheem Ashraf.
New Zealand: Kane Williamson (captain), Corey Anderson, Mark Chapman, Lockie Ferguson, Colin de Grandhomme, Adam Milne, Colin Munro, Ajaz Patel, Glenn Phillips, Seth Rance, Tim Seifert, Ish Sodhi, Tim Southee, Ross Taylor.
Company profile
Company name: Suraasa
Started: 2018
Founders: Rishabh Khanna, Ankit Khanna and Sahil Makker
Based: India, UAE and the UK
Industry: EdTech
Initial investment: More than $200,000 in seed funding
While you're here
Mustafa Alrawi: To get the 'jab' done, there must be patience and empathy
Damien McElroy: Anti-science attitudes in America are proving lethal
Editorial: What makes the UAE such a good place to test vaccines?
Editorial: The fight against Covid-19 should be guided by science
Key developments
- Singapore summit: Trump and Kim launch charm offensive as meetings begin
- Donald Trump and Kim Jong-un shake hands ahead of one-on-one discussion
- US and North Korean teams sit down for bilateral summit
- Kim: “I believe this is a good prelude for peace."
- Trump: “We will solve it, we will be successful.”
All times UTC 4
On Women's Day
Dr Nawal Al-Hosany: Why more women should be on the frontlines of climate action
Shelina Janmohamed: Why shouldn't a spouse be compensated fairly for housework?
Samar Elmnhrawy: How companies in the Middle East can catch up on gender equality
The National Editorial: Is there much to celebrate on International Women's Day 2021?
Our commentary on Brexit
- Con Coughlin: Choice of the British people will be vindicated
- Sam Williams: Departure is influenced by its sense of place
While you're here
Gavin Esler: Coronavirus offers stinging rebuke to protectionists
Kareem Shaheen: Pandemics can save us from waging wars
Sholto Byrnes: Why the climate change clash will get nastier
Martin Sabbagh profile
Job: CEO JCDecaux Middle East
In the role: Since January 2015
Lives: In the UAE
Background: M&A, investment banking
Studied: Corporate finance
How to register as a donor
1) Organ donors can register on the Hayat app, run by the Ministry of Health and Prevention
2) There are about 11,000 patients in the country in need of organ transplants
3) People must be over 21. Emiratis and residents can register.
4) The campaign uses the hashtag #donate_hope
KILLING OF QASSEM SULEIMANI
National Editorial: Suleimani has been killed, now we must de-escalate
Mina Al Oraibi: Air strike casts a long shadow over the decade ahead
Jack Moore: Why the assassination is such a monumental gamble
Matthew Levitt: Iran retains its ability to launch terror attacks
Hussein Ibish: Trump's order on solid constitutional ground
Simon Waldman: Cautious Israel keeping a low profile
Rashmee Roshan Lall: Sound of silence in South Asia
Fanar Haddad: The Iranian response will be gradual
Richard Olson: Why Afghanistan will be very wary
World Mental Health Day
Blackpink World Tour [Born Pink] In Cinemas
Starring: Rose, Jisoo, Jennie, Lisa
Directors: Min Geun, Oh Yoon-Dong
Rating: 3/5
Evacuations to France hit by controversy
- Over 500 Gazans have been evacuated to France since November 2023
- Evacuations were paused after a student already in France posted anti-Semitic content and was subsequently expelled to Qatar
- The Foreign Ministry launched a review to determine how authorities failed to detect the posts before her entry
- Artists and researchers fall under a programme called Pause that began in 2017
- It has benefited more than 700 people from 44 countries, including Syria, Turkey, Iran, and Sudan
- Since the start of the Gaza war, it has also included 45 Gazan beneficiaries
- Unlike students, they are allowed to bring their families to France
The five pillars of Islam
1.
2.
3.
4.
5.
The Sand Castle
Director: Matty Brown
Stars: Nadine Labaki, Ziad Bakri, Zain Al Rafeea, Riman Al Rafeea
Rating: 2.5/5
Neighbourhood Watch
The burning issue
The internal combustion engine is facing a watershed moment – major manufacturer Volvo is to stop producing petroleum-powered vehicles by 2021 and countries in Europe, including the UK, have vowed to ban their sale before 2040. The National takes a look at the story of one of the most successful technologies of the last 100 years and how it has impacted life in the UAE.
Read part four: an affection for classic cars lives on
Read part three: the age of the electric vehicle begins
Read part two: how climate change drove the race for an alternative
SM Town Live is on Friday, April 6 at Autism Rocks Arena, Dubai. Tickets are Dh375 at www.platinumlist.net
SPECS
MORE FROM ED HUSAIN: The UAE-Israel accord is a win for every Muslim
