Please provide your email address to receive an email when new articles are posted on . The European Medicines Agency has granted orphan drug designation to a small molecule intended to treat spinal ...
Spinal and bulbar muscular atrophy (SBMA) is a rare X-linked neuromuscular disorder that results from an abnormal expansion of gene sequences in the androgen receptor gene. SBMA has an adult onset and ...
Because bulbar muscle function was severely impaired it was necessary to continue pyridostigmine for Case 1 until about ten hours before administration. In Case 2, pyridostigmine was continued until ...
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