The Seminars/Events That We Attended:

A special event for Rare Diseases Day was held by NIH, the world’s largest biomedical research institution in USA. Along with raising awareness about these diseases, research studies and collaborations of the institution were conveyed.

The largest muscular dystrophy association of USA – MDA, organized a virtual clinical and scientific conference on the diseases of muscular dystrophy. At the conference, we had the opportunity to follow up with all the details of the latest studies on muscular dystrophy diseases.

We attended the conference held on the internet by CureCMD, which is the largest association in the world focused on CMD (KMD) diseases and with which we cooperate as CMDTR. At the conference, we had the opportunity to listen to the latest diagnosis, treatment studies and the most effective patient care recommendations for CMD (Congenital Muscular Dystrophy) diseases in all details. Prof Dr. Göknur Haliloğlu and Prof. Dr. Carsten Bönnemann from the CMDTR Scientific Advisory Board attended the conference as speakers.

We attended the conference held on the internet by NCATS, a unit of the world’s largest biomedical research institution NIH in USA. In particular, we had the opportunity to learn about the latest developments in gene therapies, which concern rare diseases, the importance of early diagnosis and how they can be applied/disseminated, and how the developed gene therapies can be delivered to everyone under equal conditions, from the most influential names.

We participated the online discussion organized by the “Mila’s Miracle” foundation, which conducts important studies on genetic diseases. Dr. Steven Gray and Dr. Wendy Chung were the speakers. Opinions were exchanged on the rapidly progressing scientific developments related to genetic disease treatments.

EURO-NMD is a European Reference Network for the thematic grouping of rare neuromuscular diseases (NMDs), a broad group of related disorders that represent a major cause of mortality and lifelong disability in children and adults. We attended the first one of the summer school webinar series. It was about the “Introduction to clinical trials”. Dr. Michela Guglieri kindly answered our questions and shared important information related to the topic. 

Certificate of Attendance Euro-NMD Webinar Sabrina Sacconi -Teresinha Evangelista 01_07_2021Onur Cakir
Cure CMD Omigapil Community Statement 2021-09-FINAL