The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.

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.

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. 

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.