Abstract: Methods for treating Phelan McDermid Syndrome (PMS) in subjects having the disorder or in subjects predisposed to develop the disorder via administration of farnesyl dibenzodiazepinone compounds are provided. The use of farnesyl dibenzodiazepinone compounds in the manufacture of a medicament for treating a subject having PMS is provided as well.
Type:
Application
Filed:
April 14, 2023
Publication date:
August 31, 2023
Applicant:
AMO Pharma Ltd.
Inventors:
Michael Snape, Patricia Cogram, Robert Deacon
Abstract: The use of 2,4-disubstituted thiadiazolidinone (TDZD) compounds, such as Tideglusib (4-Benzyl-2-naphthalen-1-yl-[1,2,4]thiadiazolidine-3,5-dione), in methods of inhibiting RNA molecules comprising abnormal trinucleotide repeats (such as CUG) in their sequences is provided. Such methods include methods of inhibiting RNA molecules having abnormal repeat sequences, as well as methods of treating and/or preventing diseases associated with the presence of RNA molecules having abnormal repeat sequences, such as myotonic dystrophy type 1 (DM1).
Abstract: The use of 2,4-disubstituted thiadiazolidinone (TDZD) compounds, such as Tideglusib (4-Benzyl-2-naphthalen-1-yl-[1,2,4]thiadiazolidine-3,5-dione), in methods of inhibiting RNA molecules comprising abnormal trinucleotide repeats (such as CUG) in their sequences is provided. Such methods include methods of inhibiting RNA molecules having abnormal repeat sequences, as well as methods of treating and/or preventing diseases associated with the presence of RNA molecules having abnormal repeat sequences, such as myotonic dystrophy type 1 (DM1).
Abstract: Methods for treating Phelan McDermid Syndrome (PMS) in subjects having the disorder or in subjects predisposed to develop the disorder via administration of farnesyl dibenzodiazepinone compounds are provided. The use of farnesyl dibenzodiazepinone compounds in the manufacture of a medicament for treating a subject having PMS is provided as well.
Type:
Application
Filed:
October 25, 2018
Publication date:
October 22, 2020
Applicant:
AMO Pharma Ltd.
Inventors:
Michael Snape, Patricia Cogram, Robert Deacon