Patent ID: 11865128
Assignee: TECHNION RESEARCH & DEVELOPMENT FOUNDATION LIMITED
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  B  C | IPC A  B  C

Claim 2:
3. A pharmaceutical composition being packaged in a packaging material and identified in print, in or on said packaging material, for use in the treatment of a genetic disorder associated with a premature stop codon mutation and/or a protein truncation phenotype, the composition comprising the compound:, or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier; and
the genetic disorder being selected from the group consisting of cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), ataxia-telangiectasia, Hurler syndrome, hemophilia A, hemophilia B, Usher syndrome, Tay-Sachs Becker muscular dystrophy (BMD), Congenital muscular dystrophy (CMD), Factor VII deficiency, Familial atrial fibrillation, Hailey-Hailey disease, McArdle disease, Mucopolysaccharidosis, Nephropathic cystinosis, Polycystic kidney disease, Rett syndrome, Spinal muscular atrophy (SMA), X-linked nephrogenic diabetes insipidus (XNDI) and X-linked retinitis pigmentosa.