Miscellaneous Preparations

4 Drugs classified under this therapeutic system

Gene Therapy. delandistrogene moxeparvovec 1.33 X 10^13.
Vial. Sol. for inf. 1.33 x 10¹³ vector genomes/ml  10-70 vials X 10 ml
For single-dose IV infusion only. Recommended dose is 1.33 × 10¹⁴ vector genomes per kilogram (vg/kg) of BW (or 10 mL/kg BW) for pts weighing less than 70 kg or 9.31 × 10¹⁵ vg total fixed dose for pts weighing 70 kg or greater. See full prescription info.
Tmt. of ambulatory ped. pts aged 4 to under 8 years with Duchenne musc. dystrophy (DMD) with a confirmed mutation in the DMD gene.
C/I: Hypersens. Any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene.

Antisense Oligonucleotide. Eteplirsen 50 mg/ml.
Vial. Conc. for sol. for inf. 1 X 100 mg/2 ml or 500 mg/10 ml
 30 mg/kg admin. once weekly
 For Duchenne muscular dystrophy (DMD) in pts who have a confirmed mutat. of the DMD gene that is amenable to exon 51 skipping
C/I: Hypersens.

C Natriuretic Peptide. vosoritide 0.4 mg, 0.56 mg, 1.2 mg.
Vial: powder and solvent for solution for inject. X 10
To be given as a daily SC inject. The recommemd. dose is based on the patient's weight and is between 15-30 μg/kg, where the higher dose is given to smallest children. See lit.
Tmt. of achondroplasia in paed. pts. whose epiphyses are not closed. The diagnosis should be confirmed by appropriate genetic testing.
C/I:  Hypersens.

Drugs for Disorders of the Musculo-Skeletal System – Enzymes. Collagenase Clostridium Histolyticum 0.9 mg/Vial.
VIAL (pwdr. for sol. for inject.): vial of 0.9
mg powder+ vial of solvent (3ml).
Dupuytren’s contracture: 0.58 mg per
inject. into a palpable Dupuytren’s cord.
See lit.
Peyronie’s disease: 0.58 mg per injection
administered into a Peyronie’s plaque.
See lit.
Tmt. of Dupuytren’s contracture in adult
pts. with a palpable cord.
The tmt. of adult men with Peyronie’s dis.
with a palpable plaque and curvature
deformity of at least 30 degrees at the
start of ther.
C/I: Hypersens. Tmt. of Peyronie’s
plaques that involve the penile urethra
due to potent. risk to this structure.