Trehalose

Published

dm+d

22209811000001107

New Medicines

CabalettaMuscular dystrophy - oculopharyngeal muscular dystrophy (OPMD)

Information

Cabaletta
New molecular entity
Seelos
Seelos

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes
Yes

Category

Protein aggregation inhibitors
OPMD affects less than 5 in 10,000 people in the EU. The disease is most often diagnosed in the fifth to sixth decade of life and progresses throughout the patients life [2].
Muscular dystrophy - oculopharyngeal muscular dystrophy (OPMD)
Intravenous