SMA Newsroom

ResearchJun 27, 2022

Poster on MANATEE: Roche and Genentech clinical study of GYM329 (RO7204239) in combination with risdiplam treatment in children with SMA

MANATEE is a global, Phase 2/3 study that will assess GYM329 in combination with risdiplam in ambulant (able to walk independently) children with SMA aged 2-10 years. Children who have been previously treated with either risdiplam, nusinersen or onasemnogene abeparvovec are eligible, as well as those who have not received treatment before. Other eligibility criteria exist. 

For more information, please see below to consult the poster.

Clinical trialsJun 22, 2022

Scholar Rock: Positive Phase 2 Topaz Trial Extension.

On June 17th, Scholar Rock announced new data from the Phase 2 TOPAZ trial extension period evaluating outcomes after 24-months of treatment.

Apitegromab, or SRK-015, is a muscle-directed therapy, developed by Scholar Rock that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. The aim of the TOPAZ clinial trial, a phase 2 study, is to evaluate the efficacy and safety of apitegromab in people with later-onset SMA. 

OtherNewslettersJun 21, 2022

Community letter from Novartis Gene Therapies June 2022

In this June 2022 community update, Novartis Gene Therapies shares the latest Gene Therapy Education Resources and provide clinical updates on completed, ongoing and upcoming Clinical Studies. 

For more information please see the link below.

SMA EventsJun 15, 2022

3rd International Scientific Congress on SMA. Deadlines for Abstract Submission and Early Registration.

1st of July: deadline for Early Registration and Abstract Submission.