In order to treat an underserved population of patients with a rare condition whose muscles gradually become weaker until they can no longer walk, a team of researchers across Japan have completed a clinical study to confirm the safety of long-term administration of a therapeutic drug.
Tohoku UniversityAug 20 2024
Despite there being a demand from patients, developing a treatment to slow down symptom progression has been difficult due to the rarity of the disease. For example, there are approximately 400 people with GNE myopathy in all of Japan." A treatment for a population this size is considered an "ultra orphan drug" - because it is not profitable for pharmaceutical companies to develop treatment for such a small group.
Related StoriesResearchers conducted investigator-initiated phase I and phase II/III studies, and an efficacy confirmation study sponsored by Nobelpharma Co., Ltd. These studies demonstrated the treatment effects of an ultra-orphan drug, "Aceneuramic Acid Extended Release Tablets 500mg" for GNE myopathy.
Distal Myopathy Efficacy Muscle Muscle Atrophy Rare Disease Vacuoles
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