top of page

Muscular Dystrophy Association (MDA) | 2023 | News | Updates | iPharmacenter


Novartis has presented the long-term follow-up data of two studies, LT-001 and LT-002, demonstrating the efficacy and durability of Zolgensma (onasemnogene abeparvovec) in children with spinal muscular atrophy.

In LT-001, children with SMA symptoms are included; in LT-002, children with presymptomatic intravenous cohort were included. The LT-001 trial is a 15-year LTFU study in which, after 7.5 years, children treated with SMA symptoms achieved all previously achieved motor milestones.


Novartis also presented the interim results of a 15-year LT-002 study, assessing the long-term efficacy of both presymptomatic and symptomatic patient populations, which were administered as intravenous (IV) and intrathecal (IT) administration. All patients maintained motor milestones. Novartis announced that most patients in the LT-002 study had not been administered add-on therapy.


Roche has presented the long-term SUNFISH study results, demonstrating the efficacy of Evrysdi (risdiplam) in people aged two to twenty-five years with spinal muscular atrophy (SMA). The increase in motor function was sustained until the fourth year, whereas the adverse events were reduced.


The function improvement was assessed using changes in Motor Function Measure 32 (MFM-32) and Revised Upper Limb Module (RULM). People with Type 2 or 3 SMA show a decline in motor function.


Roche stated that Evrysdi was well-tolerated, and the common side events were headache, fever, and upper respiratory tract infections.

Evrysdi, in combination with an anti-myostatin molecule, is in Phase 2/3 development for SMA.


0 comments
bottom of page