The online presentation event of “The STRIDE REGISTRY for Duchenne Muscular Dystrophy” organized by Parent Project in collaboration with O.M.a.R.- Rare Diseases Observatory and the unconditional contribution of PTC Therapeutics Italy, will take place on Wednesday, the 13th of January, in 2 sessions. The first session, in Italian, will be at 1 pm and the second session, in English, will be at 2 pm.
The event will be an opportunity to introduce families, clinicians and institutions to the STRIDE (Strategic Targeting of Registries and International Database of Excellence) study, the first drug registry for patients with Duchenne muscular dystrophy (DMD). The registry today represents the largest study in the world based on clinical practice (real-world) of DMD patients with nonsense mutations (nmDMD). STRIDE is an international, observational, post-approval study that has been requested by regulatory authorities for the post-marketing evaluation of the use of ataluren and is ongoing in countries where ataluren is commercially available or through an early access program.
Duchenne muscular dystrophy or DMD is a rare genetic disorder that affects relatively few people. There is little documented information on the epidemiology of the disease, in particular for the different subgroups of mutations in patients with DMD. Furthermore, it is often difficult to recruit large numbers of patients in clinical trials, and generally those recruited represent a homogeneous population with well-defined characteristics. The STRIDE registry is, therefore, important as it collects and integrates data from a larger number of nmDMD patients, over a longer period of time, which is useful for understanding real-world results for different purposes, such as the natural history of the disease. and the efficacy and safety of ataluren.
The agenda is available below:
It is possible to register for the webinar through the following link: below:https://zoom.us/webinar/register/WN_RVaX32gbQcmw8rzSjLfIqA
The STRIDE Registry brochure is available below:
A registration of the webinar will be available within a few days after the event.