Лонафарниб для пациентов с синдромом Хатчинсона-Гилфорда прогерия или прогероидной ламинопатией

Inclusion Criteria:

• Clinical diagnosis of HGPS or progeroid laminopathy by qualified medical doctor (based on common phenotype as described in Gordon 2015 and Merideth 2008). Confirmation with genetic testing is preferred but not required.
• Adequate hepatic function as defined by SGPT (ALT) and SGOT (AST) ≤ 5 times upper limit of normal range for age

Exclusion Criteria:

• Taking medications or foods that are known to be moderate or strong inducers or inhibitors of CYP3A4 or sensitive CYP3A substrates; or if a patient is taking one of these drugs and cannot safely discontinue or take an alternative drug, the dose of the inhibitor/inducer must be adjusted per the treating physician
• Taking digoxin, a P-gp substrate with a narrow therapeutic window.
• Severe renal impairment (GFR < 30 mL/min/1.73m2).
• Uncontrolled infection.
• Presence of any active clinically relevant medical condition that in the opinion of the treating physician would preclude patient from safely participating in the program.
• Pregnant or breast-feeding or plan to become pregnant while on therapy.