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Treatments Promoting AChR Clusters May Be New Therapeutic Approach in MuSK-MG

Treatments promoting the formation of acetylcholine receptor (AChR) clusters could represent a new therapeutic approach for people with MuSK-autoantibody positive myasthenia gravis (MuSK-MG), a preclinical study suggests. While the data still need to be confirmed in further studies, researchers highlighted the therapeutic potential of targeting MG-associated cell processes, instead of…

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.