Browsing: Conditions

For more than a decade, chimeric antigen receptor T-cell (CAR T) therapy has transformed the treatment landscape for certain blood cancers. Now, researchers believe the next major leap might come from combining CAR T with powerful gene-editing technologies such as CRISPR (which stands for clustered regularly interspaced short palindromic repeats) and TALEN (which stands for…

When Mila Makovec was three years old, her parents started to notice that something was wrong. Previously an active and healthy toddler, Mila began to get stuck on words, have sight problems, and walk strangely. This was just the start of the symptoms of her ultra-rare genetic disease, a fatal neurological disorder called Batten disease…

Becky Quick is a well-known television personality: she’s the co-anchor of the morning show Squawk Box on CNBC. From Davos to Wall Street, Quick has interviewed a stellar list of billionaires, CEOs, analysts, and investors, never seeking to inject herself into the news

Although each rare disease affects only a small number of people, collectively they represent one of the world’s largest unmet healthcare challenges. More than 7,000-8,000 rare diseases have been identified globally, yet effective treatments exist for only around 5-10 per cent of them, according to IQVIA. Together, these conditions affect an estimated 350-475 million people…