A drug to treat distal myopathy – a rare condition characterized by progressive weakness of the distal muscles of the hands and feet, leaving patients unable to walk within about a decade – was recently approved globally for use in Japan for the first time . after 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 ultra-orphan drug to be developed – a drug used to treat an exceptionally rare disease with fewer than 1,000 patients in Japan, with little opportunity to to make a profit. . It was the joint efforts of the research team and a patient association that paved the way for this achievement.
“We could present a model case from a university that is leading the development of a drug for a rare disease,” said Masashi Aoki, professor of neurology at Tohoku University Graduate School of Medicine, who led the research team.
