Children are disproportionately affected by rare diseases, and few sufferers make it past their fifth birthday.
Thankfully, today, there are more life-saving treatment opportunities than ever before. However, the challenges of applying them effectively are immense, Prof Claire Booth told The National.
The path to effective care is often innovative or groundbreaking and there are no guides to rely on, said Booth.
“When a child is going to be the first person in the world who gets this treatment, it’s a difficult decision for the families, even though we think it’s going to work,” said Booth, a paediatric consultant at Great Ormond Street Hospital.
She is a world expert in her field and leads clinical research at the London hospital where the Zayed Centre for Research into Rare Disease in Children - which includes an outpatient facility and a laboratory for cell and gene therapy - has recently been granted expanded treatment capabilities.
Zayed Centre for Research — which opened in 2019 — is dedicated to treating the world’s rarest diseases in children, from babies born without functioning immune systems to liver and metabolic disorders. It was built partly with a £60 million donation from Sheikha Fatima bint Mubarak, and is a partnership between Great Ormond Street Hospital, University College London and Great Ormond Street Hospital Children's Charity.
“They’re such devastating diseases that they tend to present themselves early in life,” said Booth, who revealed her team's work is, for most patients, their last hope to find a cure.
“A lot of families contact us directly,” said Booth. “The standard treatment [for their child’s condition] isn’t working, or, in some cases, there is no treatment.”
But recent breakthroughs in the field mean that more children with rare diseases will survive.
Cell and gene therapy was pioneered at Great Ormond Street Hospital, a little more than 20 years ago. It involves manipulating the genes, cells or tissues causing the disease, and serves as an alternative to a bone marrow transplant for some conditions.
“When people are born with a genetic condition that leads to a disease, it's usually because a gene in their DNA isn't working properly. Gene therapy is either repairing or replacing that gene,” said Booth, who specialises in genetic disorders affecting the immune and metabolic systems.
The Zayed Centre for Research was awarded a licence to manufacture drugs by the Medicines and Healthcare Products Regulatory Authority (MHRA) in February. Stem and gene therapy products were previously produced at two labs in another wing of the hospital but the new approvals will serve to accelerate the production of medicines and increase the number of clinical trials taking place across the UK.
Research has begun on new methods of gene therapy such as in vivo, where a virus containing the corrective gene is injected directly into the body. “We try and correct the cells while they're still inside the patient,” said Booth.
In vivo treatments are currently being used for liver, brain and neuromuscular disorders as well as some metabolic conditions. “We can inject the virus directly into the fluid inside the brain,” said Booth.
Inside the 140-bench laboratory — one of the largest in Europe — Booth’s team of researchers work on different stages of an experimental treatment. “We use samples of patients’ blood cells to test the therapies that we’re developing,” said Booth.
Large glass walls on the ground floor give visitors a bird’s eye view of the basement laboratory. This emphasises the hospital’s “bench to bedside” approach, which enables a close connection between patients and clinical researchers.
“As patients come in they can see the scientists and researchers working on something that will impact them,” said Booth.
The track record of the facility is already stellar. In 2021, a clinical trial for ADA-SCIDS, an inherited disease where babies are born without a functioning immune system, found that 48 of 50 patients no longer showed symptoms of the disorder after a single round of treatment.
Previously, patients with ADA-SCIDS had to have twice or weekly injections until they could find a donor for a bone marrow transplant. “All the patients are alive, and 96 per cent of them were essentially cured with a one-off gene therapy,” said Booth of the trial done in collaboration with the University of California, Los Angeles.
The centre’s Gene and Cell Therapy Facility is where the medicines are prepared in a delicate and fragile process overseen by a dedicated team working to tight timeframes. The facility head, Dr Barry Flutter told The National that medicines are designed to treat individual patients and have a shelf life of a few hours, making on-site manufacturing essential.
“We build on the research programmes of the hospital, and translate them into products which can be given to patients,” said Dr Flutter.
Inside, seven highly-protected and sterile laboratories are used for all stages of the preparation, and have increased capacity to manufacture gene therapy products by more than 300 per cent — from 40 products a year to 150, the centre estimates.
The simulation room, which is identical to the other labs, is used for early manufacturing stages and training.
“Now that we have the licence, we’re moving,” he said. Three of the labs, he explained, will be used to produce vectors — which are the vehicles used to transmit the working gene into the living cell — in partnership with Virocell Biologics, a commercial company. This will allow scientists to double the number of vectors the company currently produces for UK-wide clinical trials.
This will allow scientists to double the number of vectors currently produced, and will serve to increase the number of clinical trials taking place across the UK.
Making each treatment can take up to six months, Flutter explained, but there are signs that this process could become easier in the future.
In December, the hospital announced it had treated a patient with a rare “incurable” form of leukaemia, using the latest gene-editing technology. The 13-year-old patient was in remission one year after treatment, having previously been given just months to live.
Annie Etuk, a scientist at the facility, explained that the technology could allow them to progress from the delicate task of making medicines uniquely designed for each patient, to creating “off-the-shelf” products for a wider range of people.
Despite these breakthroughs, gene and cell therapy research faces tremendous challenges. “For every patient that we do treat, there are many others that aren't eligible,” said Etuk.
Rare diseases struggle to get commercial investment. “The patient population is very small, we’re talking about 10 to 20 patients being treated in the world per year” said Booth. “So companies are pulling out.”
Booth added that their belief is misguided: “What you learn in rare diseases can often be applied to much more common diseases, and this can impact huge populations of patients.” This includes treatment for cancers and sickle cell anaemia.
The AGORA initiative, founded by Booth in September last year, explores not-for-profit and sustainable ways to increase access to rare disease treatments.
Among these are the commercial partnerships afforded by the MHRA licence. “The financial sustainability of that model with help the research here at Great Ormond Street,” said Booth.
Staff shortages within the NHS remain an issue, particularly when making medicines. “It can be quite challenging to find sufficient staff to manufacture your product from start to finish,” said Etuk.
Misinformation about gene and cell therapy is also a risk that researchers are assessing. Booth recalled a recent protest outside a summit that she attended in London, triggered by a Chinese trial which announced the successful gene editing of embryos.
“There’s a level of education and awareness that needs to change. We’re changing the genetic make-up of a person’s blood, liver or brain system where the diseases are,” she said, “not their genetic make-up or family line.”
To date, hesitancy about gene therapy has not been an issue for families seeking help from the London hospital. “The families come to us with a great deal of hope,” said Booth, “because they are looking for a cure.”
If you go
The flights
The closest international airport for those travelling from the UAE is Denver, Colorado. British Airways (www.ba.com) flies from the UAE via London from Dh3,700 return, including taxes. From there, transfers can be arranged to the ranch or it’s a seven-hour drive. Alternatively, take an internal flight to the counties of Cody, Casper, or Billings
The stay
Red Reflet offers a series of packages, with prices varying depending on season. All meals and activities are included, with prices starting from US$2,218 (Dh7,150) per person for a minimum stay of three nights, including taxes. For more information, visit red-reflet-ranch.net.
What is the definition of an SME?
SMEs in the UAE are defined by the number of employees, annual turnover and sector. For example, a “small company” in the services industry has six to 50 employees with a turnover of more than Dh2 million up to Dh20m, while in the manufacturing industry the requirements are 10 to 100 employees with a turnover of more than Dh3m up to Dh50m, according to Dubai SME, an agency of the Department of Economic Development.
A “medium-sized company” can either have staff of 51 to 200 employees or 101 to 250 employees, and a turnover less than or equal to Dh200m or Dh250m, again depending on whether the business is in the trading, manufacturing or services sectors.
More from Neighbourhood Watch:
Honeymoonish
%3Cp%3E%3Cstrong%3EDirector%3A%3C%2Fstrong%3E%20Elie%20El%20Samaan%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EStarring%3A%20%3C%2Fstrong%3ENour%20Al%20Ghandour%2C%20Mahmoud%20Boushahri%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3ERating%3A%3C%2Fstrong%3E%203%2F5%3C%2Fp%3E%0A
Tamkeen's offering
- Option 1: 70% in year 1, 50% in year 2, 30% in year 3
- Option 2: 50% across three years
- Option 3: 30% across five years
Dubai Bling season three
Cast: Loujain Adada, Zeina Khoury, Farhana Bodi, Ebraheem Al Samadi, Mona Kattan, and couples Safa & Fahad Siddiqui and DJ Bliss & Danya Mohammed
Rating: 1/5
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
UAE currency: the story behind the money in your pockets
Star%20Wars%3A%20Ahsoka%20
%3Cp%3E%3Cstrong%3EDirector%3A%3C%2Fstrong%3E%20Various%20%3Cbr%3E%3Cstrong%3EStarring%3A%3C%2Fstrong%3E%20Rosario%20Dawson%2C%20Natasha%20Liu%20Bordizzo%2C%20Lars%20Mikkelsen%20%3Cbr%3E%3Cstrong%3ERating%3A%3C%2Fstrong%3E%204%2F5%0D%3Cbr%3E%3C%2Fp%3E%0A
The specs
Engine: 2.0-litre 4cyl turbo
Power: 261hp at 5,500rpm
Torque: 405Nm at 1,750-3,500rpm
Transmission: 9-speed auto
Fuel consumption: 6.9L/100km
On sale: Now
Price: From Dh117,059
Yuki Means Happiness
Alison Jean Lester
John Murray
NO OTHER LAND
Director: Basel Adra, Yuval Abraham, Rachel Szor, Hamdan Ballal
Stars: Basel Adra, Yuval Abraham
Rating: 3.5/5
Manchester City 4
Otamendi (52) Sterling (59) Stones (67) Brahim Diaz (81)
Real Madrid 1
Oscar (90)
Look%20Both%20Ways
%3Cp%3EDirector%3A%20Wanuri%20Kahiu%3Cbr%3EStars%3A%20Lili%20Reinhart%2C%20Danny%20Ramirez%2C%20David%20Corenswet%2C%20Luke%20Wilson%2C%20Nia%20Long%3Cbr%3ERating%3A%203%2F5%3Cbr%3E%3C%2Fp%3E%0A
The specs
Engine: 2.0-litre four-cylinder turbo
Power: 178hp at 5,500rpm
Torque: 280Nm at 1,350-4,200rpm
Transmission: seven-speed dual-clutch auto
Price: from Dh209,000
On sale: now
Moral education needed in a 'rapidly changing world'
Moral education lessons for young people is needed in a rapidly changing world, the head of the programme said.
Alanood Al Kaabi, head of programmes at the Education Affairs Office of the Crown Price Court - Abu Dhabi, said: "The Crown Price Court is fully behind this initiative and have already seen the curriculum succeed in empowering young people and providing them with the necessary tools to succeed in building the future of the nation at all levels.
"Moral education touches on every aspect and subject that children engage in.
"It is not just limited to science or maths but it is involved in all subjects and it is helping children to adapt to integral moral practises.
"The moral education programme has been designed to develop children holistically in a world being rapidly transformed by technology and globalisation."
The%20specs%20
%3Cp%3E%3Cstrong%3EEngine%3A%20%3C%2Fstrong%3E2.0-litre%204cyl%20turbo%0D%3Cbr%3E%3Cstrong%3EPower%3A%20%3C%2Fstrong%3E261hp%20at%205%2C500rpm%0D%3Cbr%3E%3Cstrong%3ETorque%3A%20%3C%2Fstrong%3E400Nm%20at%201%2C750-4%2C000rpm%0D%3Cbr%3E%3Cstrong%3ETransmission%3A%20%3C%2Fstrong%3E7-speed%20dual-clutch%20auto%0D%3Cbr%3E%3Cstrong%3EFuel%20consumption%3A%20%3C%2Fstrong%3E10.5L%2F100km%0D%3Cbr%3E%3Cstrong%3EOn%20sale%3A%20%3C%2Fstrong%3ENow%0D%3Cbr%3E%3Cstrong%3EPrice%3A%20%3C%2Fstrong%3EFrom%20Dh129%2C999%20(VX%20Luxury)%3B%20from%20Dh149%2C999%20(VX%20Black%20Gold)%3C%2Fp%3E%0A
Six tips to secure your smart home
Most smart home devices are controlled via the owner's smartphone. Therefore, if you are using public wi-fi on your phone, always use a VPN (virtual private network) that offers strong security features and anonymises your internet connection.
Keep your smart home devices’ software up-to-date. Device makers often send regular updates - follow them without fail as they could provide protection from a new security risk.
Use two-factor authentication so that in addition to a password, your identity is authenticated by a second sign-in step like a code sent to your mobile number.
Set up a separate guest network for acquaintances and visitors to ensure the privacy of your IoT devices’ network.
Change the default privacy and security settings of your IoT devices to take extra steps to secure yourself and your home.
Always give your router a unique name, replacing the one generated by the manufacturer, to ensure a hacker cannot ascertain its make or model number.
Kamindu Mendis bio
Full name: Pasqual Handi Kamindu Dilanka Mendis
Born: September 30, 1998
Age: 20 years and 26 days
Nationality: Sri Lankan
Major teams Sri Lanka's Under 19 team
Batting style: Left-hander
Bowling style: Right-arm off-spin and slow left-arm orthodox (that's right!)
Diriyah%20project%20at%20a%20glance
%3Cp%3E-%20Diriyah%E2%80%99s%201.9km%20King%20Salman%20Boulevard%2C%20a%20Parisian%20Champs-Elysees-inspired%20avenue%2C%20is%20scheduled%20for%20completion%20in%202028%3Cbr%3E-%20The%20Royal%20Diriyah%20Opera%20House%20is%20expected%20to%20be%20completed%20in%20four%20years%3Cbr%3E-%20Diriyah%E2%80%99s%20first%20of%2042%20hotels%2C%20the%20Bab%20Samhan%20hotel%2C%20will%20open%20in%20the%20first%20quarter%20of%202024%3Cbr%3E-%20On%20completion%20in%202030%2C%20the%20Diriyah%20project%20is%20forecast%20to%20accommodate%20more%20than%20100%2C000%20people%3Cbr%3E-%20The%20%2463.2%20billion%20Diriyah%20project%20will%20contribute%20%247.2%20billion%20to%20the%20kingdom%E2%80%99s%20GDP%3Cbr%3E-%20It%20will%20create%20more%20than%20178%2C000%20jobs%20and%20aims%20to%20attract%20more%20than%2050%20million%20visits%20a%20year%3Cbr%3E-%20About%202%2C000%20people%20work%20for%20the%20Diriyah%20Company%2C%20with%20more%20than%2086%20per%20cent%20being%20Saudi%20citizens%3Cbr%3E%3C%2Fp%3E%0A
Terror attacks in Paris, November 13, 2015
- At 9.16pm, three suicide attackers killed one person outside the Atade de France during a foootball match between France and Germany
- At 9.25pm, three attackers opened fire on restaurants and cafes over 20 minutes, killing 39 people
- Shortly after 9.40pm, three other attackers launched a three-hour raid on the Bataclan, in which 1,500 people had gathered to watch a rock concert. In total, 90 people were killed
- Salah Abdeslam, the only survivor of the terrorists, did not directly participate in the attacks, thought to be due to a technical glitch in his suicide vest
- He fled to Belgium and was involved in attacks on Brussels in March 2016. He is serving a life sentence in France
Key figures in the life of the fort
Sheikh Dhiyab bin Isa (ruled 1761-1793) Built Qasr Al Hosn as a watchtower to guard over the only freshwater well on Abu Dhabi island.
Sheikh Shakhbut bin Dhiyab (ruled 1793-1816) Expanded the tower into a small fort and transferred his ruling place of residence from Liwa Oasis to the fort on the island.
Sheikh Tahnoon bin Shakhbut (ruled 1818-1833) Expanded Qasr Al Hosn further as Abu Dhabi grew from a small village of palm huts to a town of more than 5,000 inhabitants.
Sheikh Khalifa bin Shakhbut (ruled 1833-1845) Repaired and fortified the fort.
Sheikh Saeed bin Tahnoon (ruled 1845-1855) Turned Qasr Al Hosn into a strong two-storied structure.
Sheikh Zayed bin Khalifa (ruled 1855-1909) Expanded Qasr Al Hosn further to reflect the emirate's increasing prominence.
Sheikh Shakhbut bin Sultan (ruled 1928-1966) Renovated and enlarged Qasr Al Hosn, adding a decorative arch and two new villas.
Sheikh Zayed bin Sultan (ruled 1966-2004) Moved the royal residence to Al Manhal palace and kept his diwan at Qasr Al Hosn.
Sources: Jayanti Maitra, www.adach.ae
Naga
%3Cp%3E%3Cstrong%3EDirector%3A%C2%A0%3C%2Fstrong%3EMeshal%20Al%20Jaser%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EStarring%3A%C2%A0%3C%2Fstrong%3EAdwa%20Bader%2C%20Yazeed%20Almajyul%2C%20Khalid%20Bin%20Shaddad%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3ERating%3A%20%3C%2Fstrong%3E4%2F5%3C%2Fp%3E%0A
Global state-owned investor ranking by size
|
1.
|
United States
|
|
2.
|
China
|
|
3.
|
UAE
|
|
4.
|
Japan
|
|
5
|
Norway
|
|
6.
|
Canada
|
|
7.
|
Singapore
|
|
8.
|
Australia
|
|
9.
|
Saudi Arabia
|
|
10.
|
South Korea
|
MOUNTAINHEAD REVIEW
Starring: Ramy Youssef, Steve Carell, Jason Schwartzman
Director: Jesse Armstrong
Rating: 3.5/5
COMPANY%20PROFILE
%3Cp%3E%3Cstrong%3EName%3A%3C%2Fstrong%3E%20Carzaty%2C%20now%20Kavak%3Cbr%3E%3Cstrong%3EBased%3A%3C%2Fstrong%3E%20Dubai%3Cbr%3E%3Cstrong%3ELaunch%20year%3A%20%3C%2Fstrong%3ECarzaty%20launched%20in%202018%2C%20Kavak%20in%20the%20GCC%20launched%20in%202022%3Cbr%3E%3Cstrong%3ENumber%20of%20employees%3A%3C%2Fstrong%3E%20140%3Cbr%3E%3Cstrong%3ESector%3A%3C%2Fstrong%3E%20Automotive%3Cbr%3E%3Cstrong%3EFunding%3A%20%3C%2Fstrong%3ECarzaty%20raised%20%246m%20in%20equity%20and%20%244m%20in%20debt%3B%20Kavak%20plans%20%24130m%20investment%20in%20the%20GCC%3C%2Fp%3E%0A
The%20specs
%3Cp%3E%3Cstrong%3EEngine%3A%3C%2Fstrong%3E%20Dual%20synchronous%20electric%20motors%0D%3Cbr%3E%3Cstrong%3EPower%3A%20%3C%2Fstrong%3E660hp%0D%3Cbr%3E%3Cstrong%3ETorque%3A%20%3C%2Fstrong%3E1%2C100Nm%0D%3Cbr%3E%3Cstrong%3ETransmission%3A%20%3C%2Fstrong%3ESingle-speed%20automatic%0D%3Cbr%3E%3Cstrong%3ETouring%20range%3A%20%3C%2Fstrong%3E488km-560km%0D%3Cbr%3E%3Cstrong%3EPrice%3A%20%3C%2Fstrong%3EFrom%20Dh850%2C000%20(estimate)%0D%3Cbr%3E%3Cstrong%3EOn%20sale%3A%20%3C%2Fstrong%3EOctober%3C%2Fp%3E%0A
