Tags : Spinal Muscular Atrophy


Chugai Reports NDA Submission of Risdiplam to the MHLW as

Shots: The NDA submission is based on FIREFISH study assessing Risdiplam in infants with symptomatic SMA Type 1 & SUNFISH study in children and young adults with SMA Type 2 or 3 FIRESISH study results: improvement in survival and motor milestones in infants. SUNFISH study results: improvement in motor function in people aged 2-25 with […]Read More


Novartis’ Zolgensma (onasemnogene abeparvovec) Receives MHLW’s Approval for Spinal Muscular

Shots: The approval is based on P-I START, START long-term follow-up, P-III STR1VE-US, P-III SPR1NT and P-I/II STRONG (intrathecal). START and STR1VE-US assess Zolgensma (one-time, IV infusion) in symptomatic SMA Type 1 patients aged <6mos. with one/two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene respectively The P-III SPR1NT […]Read More


Roche Reports Results of Risdiplam in SUNFISH Part 2 Study

Shots: The SUNFISH part 2 study involves assessing risdiplam vs PBO in patients aged 2-25yrs. with non-ambulatory type 2 or 3 SMA The SUNFISH part 2 study resulted in meeting its 1EPs & 2EPs i.e. greater change from baseline in MFM-32 & improvement in RULM @12mos., strongest responses in MFM-32 in younger patients aged 2-5 […]Read More