Neonatal diabetes, a rare but severe form of the disease, used to mean a life of daily insulin injections and strict diet monitoring, often from birth; now, however, many patients can take well known drugs called sulphonylureas instead. This transformation in their lives is due to the research of Professors Andrew Hattersley and Frances Ashcroft, who found that the condition was caused by a genetic mutation disrupting a critical potassium channel involved in the insulin secreting pathway and that sulphonylurea drugs could restore the channels normal behaviour.
Lovely to hear the impact the research is having for real people - great film.
therealdealdoc 2 years ago
It is now known that there are a number of different genetic mutations that can cause permanent neonatal diabetes; many do not respond to sulfonylurea drugs. For those who do, it is indeed life-changing.
JudithIN410 2 years ago 2