Genetics

New taxonomy of non-skeletal rare disorders with impact on bone

Thanks to major progress in the understanding and management of rare congenital diseases and syndromes, many patients with these rare disorders are now living longer lives. With this progress it has become apparent that many ...

Genetics

CRISPR provides hope of sickle cell cure

Thanks to the 'cut and paste' gene editing technique CRISPR, scientists are homing in on a cure for sickle cell disease—a genetic blood disorder—while other research is looking at how to expand the potential of CRISPR-based ...

Other

US and Gates Foundation plan $200m for sickle cell, HIV cures

The US government and the Bill and Melinda Gates Foundation pledged Wednesday to jointly invest $200 million over the next four years to achieve affordable gene therapy-based cures for sickle cell disease (SCD) and HIV.

Diseases, Conditions, Syndromes

ATS publishes new clinical guideline on home oxygen for children

The American Thoracic Society has developed a new clinical practice guideline for home oxygen therapy for children. The guideline appears in the Feb. 1 edition of the Society's American Journal of Respiratory and Critical ...

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