WDO Member Meeting
The main aim of the WDO Member Meeting was to gather patient organizations, scientists, researchers and experts working in the Duchenne field to share and build on each other’s knowledge to create innovative ideas and practical development experience on Duchenne and Becker muscular dystrophy. Over 50 participants from 24 countries were represented during the meeting that took place in Rome last February 15th.
Prof Dr. Annemieke Aartsma-Rus and Prof. Dr. Nathalie Goemans hosted two mini-masterclasses on preclinical and clinical drug development. In the discussion, that was moderated by chaired by Prof. Dr. Eugenio Mercuri, it was found to be imperative that patient organizations are a credible source of information on clinical trials and only support optimal trials. Following the mini-masterclasses, Dr. Jarod Wong gave a presentation on endocrinology and bone health.
“By sharing data, dialogue with regulators to gain qualification from CHMP and harmonization in IT support we can overcome the rare disease problem and empower trials” – Prof. Dr. Nathalie Goemans.
In the afternoon, companies joined the meeting to attend several presentations concerning the collection and sharing of patient data by Pat Furlong of PPMD USA and Elizabeth Vroom from WDO. In addition to the clinical development updates, Dr. Ir. Joan Lobo Prat and Dr. Mariska Jansen provided the members with an update on technological developments, considering assistive and rehabilitation devices. This part was particularly perceived to be extremely valuable, since these devices are beneficial to all Duchenne and Becker affected, regardless of which mutation.