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Breakthrough for baby with rare illness in world-first gene therapy trial
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21st March 2025 - The Independent
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A baby boy with a rare genetic disorder is making “incredible” progress after he became the first infant to receive a groundbreaking new gene therapy.
Fourteen-month-old Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was a few weeks old - becoming just one of 15 each year diagnosed with the rare genetic disease.
An OTC deficiency is when ammonia, a waste product that is generated when the body breaks down proteins, builds up in a person’s blood.
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Ammonia is usually processed in the liver and removed through urine but patients with the condition have a genetic deficiency in the protein in the liver which is responsible for detoxifying ammonia, allowing ammonia to build up, which can be toxic. It can cause life-threatening complications including brain damage, coma or seizures, if left untreated.
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NHS England Job Cuts: A Cost-Saving Strategy or a Dangerous Gamble?
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The NHS is facing one of its most radical restructures in history. Sir Jim Mackey, NHS England’s incoming interim chief executive, has announced plans to “radically reduce and reshape” the organisation, potentially cutting the workforce by half. This follows NHS England’s recent 15% reduction, equating to 2,000 job losses to save £325 million.
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The justification? Streamlining operations, cutting duplication, and ensuring the “best possible use of taxpayers’ money.” But for frontline staff and patients, the key question is: will this lead to a more efficient system, or is it a short-sighted cost-cutting exercise that worsens the healthcare crisis?
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A Workforce Under Strain
Reducing bureaucracy may sound beneficial, but the NHS isn’t suffering from excess management—it’s suffering from unmet demand.