Gene therapy improves vision in rare hereditary blindness condition
A phase I clinical trial in the US has successfully used gene therapy to improve vision in individuals suffering from a rare form of hereditary blindness. The promising results of this trial pave the way for future trials and may eventually lead to a cure for several forms of congenital blindness...
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The problem with 21st Century Motherhood
by Dr Sammy Lee
Did the death of Maria Bousada change public attitudes to the modern phenomenon headlined as 'Oldest Mums'? The world's media certainly made hay and the news reverberated for a few days; and it seems likely that the Channel 4 documentary 'the Worlds Oldest Mums' was rescheduled to screen early to catch the media wave which the death generated. The aftermath, though, of this tsunami seems to have largely been relative indifference....
The case for integrated service delivery in the UK for patients with rare diseases
by Alastair Kent
Rare Disease UK is a national alliance of key stakeholders including patient organisations, industry, clinicians, academics and individuals bought together to call for a response to the unmet care needs of those with rare diseases who currently struggle to get access to integrated care and support from the National Health Service (NHS). There are over 6000 rare diseases affecting over 3.5 million people (one in 17) in the UK...