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doi: 10.1038/537s148apmid: 27652780
Problems with the lysosome cause more than just lysosomal storage disorders. This crucial cellular component has a surprising role in several common and complex conditions.
doi: 10.1038/537s146apmid: 27652779
Lysosomal storage disorders (LSDs) highlight the diverse ways in which the failure of a single organelle can bring cells to their knees. Most are rare and poorly understood, making the development of therapies a daunting task.
doi: 10.1038/537s154apmid: 27652783
Treatments that can cross the blood–brain barrier are needed if doctors are to treat the devastating neurological symptoms of many lysosomal storage disorders.
doi: 10.1038/537s152apmid: 27652782
Most rare diseases lack even one approved treatment. Regulators have tried to encourage drug development, but advocacy groups are having to fight to get the research done.
Futerman, Anthony H.; Hardy, John
doi: 10.1038/537s160apmid: 27652785
Two competing hypotheses about Parkinson's and Gaucher's diseases need to be knitted together, say Anthony H. Futerman and John Hardy.
doi: 10.1038/537s158apmid: 27652784
In the 1980s, bone-marrow transplants transformed the lives of children with fatal lysosomal diseases. Researchers are now working on another revolutionary treatment.
doi: 10.1038/537s162apmid: 27652786
It is now feasible to test babies for several lysosomal storage disorders, but this goes against the gold standard for screening that was established nearly 50 years ago. The ethical issues raised are forcing a rethink of the way that newborns are screened.
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