A drug to treat distal myopathy — a rare disorder characterized by progressive weakness of the distal muscles of the hands and feet, with patients losing the ability to walk in roughly 10 years — was recently approved for the first time worldwide, for use in Japan, following clinical trials at Tohoku University in Sendai.
The disease is estimated to affect only 300 to 400 people in Japan, and it is rare for an ultraorphan drug — a drug used to treat an exceptionally rare disease with fewer than 1,000 patients in Japan, with little possibility of making profits — to be developed. It was the united efforts of the research team and a patients’ association that paved the way for that achievement.
“We could present a model case of a university leading development of a drug for a rare disease,” said Masashi Aoki, professor of neurology at Tohoku University Graduate School of Medicine, who headed the research team.