APPLICATION OF PEGILIATED IGF-I OPTIONS FOR TREATMENT OF NEUROMUSCULAR DISORDERS

摘要

1. The use of a polyethylene glycol- (PEG) or IGF-I variant variant for producing a pharmaceutical composition for treating, preventing and / or slowing a neuromuscular disorder, wherein said polyethylene glycol- (PEG) or IGF-I variant variant is characterized in that it is derived from an amino acid sequence human wild-type IGF-I (SEQ ID NO: 1) and carries one or two amino acid changes at amino acid positions 27, 65 and 68, so that one or two amino acids at positions 27, 65 and 68 are polar amino acids the other, but not lysine and PEG is attached to at least one lysine residue. ! 2. The use according to claim 1, where the pegylated variant of IGF-I is characterized by the following amino acid changes in the amino acid sequence of the wild-type human IGF-I (SEQ ID NO: 1):! (a) K65R and K68R (SEQ ID NO: 2)! (b) K27R and K68R (SEQ ID NO: 3) or! (c) K27R and K65R (SEQ ID NO: 4). ! 3. The use according to claim 1, where the indicated pegylated variant of IGF-I is mono-pegylated at K68 and is characterized by the following amino acid changes in the amino acid sequence of wild-type human IGF-I (SEQ ID NO: 1):! K27R and K65R (SEQ ID NO: 4). ! 4. The use according to claim 1, where the specified PEG has a total molecular weight of from 20 to 100 kDa. ! 5. The use according to claim 1, where the specified pegylated variant of IGF-I is additionally pegylated at the N-terminal amino acid. ! 6. The use according to claim 1, where the specified pegylated variant of IGF-I is mono-pegylated either at K65 or at K68, or is diphegylated at K65 and K68. ! 7. The use according to claim 1, wherein said pegylated variant of IGF-I is characterized in that up to three amino acids are shortened at the N-terminus. ! 8. П�