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ACS drug gets orphan designation |
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A respiratory drug currently undergoing phase II clinical trials in the US has been awarded orphan drug designation from the country's Food and Drug Administration.
Anthera Pharmaceuticals announced that A-001, which is being studied for is effectiveness in preventing acute chest syndrome (ACS) in patients living with blood disorder sickle cell disease (SCD), also won approval for use in expanded tests.
SCD patients aged five and older will now be eligible to take part in trials of the drug, which it is hoped will uncover a new treatment option for people who develop ACS.
The condition can cause severe damage to the lungs and sometimes results in death.
It can pose a significant problem to patients and their doctors, with experts from Boston University's Translational-Clinical Research team estimate that up to 40 per cent of people diagnosed with SCD suffer from ACS at some point in their lives.
Masters is a global healthcare solutions provider which can source orphan drugs and medicines still undergoing clinical trials from manufacturers around the world for patients with a unique unmet need. |