PTC Therapeutics has reported results from Part 2 of the pivotal FIREFISH trial of Evrysdi (risdiplam), which demonstrated that infants with type 1 spinal muscular atrophy (SMA) continued to show improvements in survival and key motor milestones after two years of treatment. PTC Therapeutics has reported results from Part 2 of the pivotal FIREFISH trial
“Thanks to this technology and to the specific fighting capabilities of these cells, extraordinary results have been obtained: The therapy is a new hope for patients who no longer responded to other treatments. The therapy seems to have a long-lasting effect after a single application Unlike many other therapies, which benefit only a small portion
The CLI study is unlikely to meet its primary endpoint by the time of the final analysis Substantial low number of events in the placebo group in the CLI study decreased the statistical power of the study to meet its primary endpoint PLX-PAD was well tolerated, and no safety concerns were raised Company to terminate the CLI study to focus on different therapeutic areas in its pipeline and expects three clinical readouts within the coming calendar year
Conventional wisdom has it that too many cooks spoil the broth. But such judiciousness is not usually considered the basis for scientific pronouncements or international rulings on the need to limit the number of people in kitchens. But, when it comes to video games, conventional wisdom, not science, forms the basis for our thinking and
The RESTORE position paper represents the cumulative efforts of the RESTORE community, and the steering committee since the start of the RESTORE initiative in 2019. It outlines, in depth, the RESTORE view on why Europe should invest in Advanced Therapies, the roadblocks in Advanced Therapy development and implementation into patient care, and makes recommendations about
Chimeric antigen receptor (CAR) technology and its application to regulatory T cells (Tregs) has garnered interest among researchers in the field of cell and gene therapy. Merging the benefits of CAR technology with Tregs offers a novel and promising therapeutic option for durable reshaping of undesired immune responses following solid organ or hematopoietic stem cell
Despite the first clinical studies reporting on a correlation betweenthe migration potential of transplanted stem cells and their therapeutic efficacy, it is unclear if effective stem cell therapies requirereliable and sufficient delivery of the cells to the desired anatomiclocations, as well as survival of the cellular graft. Moreover, the many complex processes involved in stem
In the early days of COVID-19, the Alliance for Regenerative Medicine (ARM) was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space. “It was a really specific time when the world and the markets were clearly reeling from the first appreciation for the seriousness of COVID-19,” Janet
The Alliance for Regenerative Medicine (ARM) welcoming the opportunity to provide feedback on the roadmap to the EC pharmaceutical strategy has sent the following response. ARM is the leading international advocacy organisation dedicated to realizing the promise of advanced therapy medicinal products (ATMPs) and represents the interests of 350+ members worldwide and 70+ members across
Mayzent (siponimod) has been approved by the European Commission (EC) for adults with secondary progressive multiple sclerosis (SPMS) based on the results of the EXPAND trial. The European Commission (EC) has approved Mayzent (siponimod) for the treatment of adults with secondary progressive multiple sclerosis (SPMS) with active disease evidenced by relapses or imaging features of inflammatory activity. Approximately 80
