Browsing: rare

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When most people think about places advancing rare disease diagnostics, sequencing, and treatments, big U.S. centers like Rady Children’s Institute for Genomic Medicine or Boston Children’s Hospital come to mind. But clinician scientists Vorasuk Shotelersuk, MD, and Shahida Moosa, MD, PhD, are showing what rare disease genomics can look like when it is driven from…

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…

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…