10th international congress on the occasion of Rare Disease Day by believing in the theme ‘’RARE but not ALONE!’’ in order to sympathy, supporting rare patients as well as presenting RADOIR’s lastest scientific, training and social achievements which will be held at Razi International Conference Center (RICC)on the March 2nd , 2019.
By Ed Miseta, Chief Editor, Clinical Leader
Patient recruitment has always been challenge for companies conducting clinical trials. Knowing how to properly engage with advocacy groups can determine whether they will treat you as a trusted partner or a necessary evil.
In order to establish R & D collaborative committee on the scientific research prioritizations for the rare patients’ needs, a meeting session was held together with RADOIR & TUMS authorities.
Rare Diseases Foundation of Iran and ROYAN Institute held a meeting session on the means of scientific research collaboration in the field of rare diseases and genetic disorders on Wednesday 2nd Jan, 2019.
Health socialization & social responsibility on health care system is the main topic of discussions during Dr.Hashemi’s ministership which was completely changed through emphasizing on NGOs’ impressive role, accordingly.
Using both CRISPR-Cas9 and base editor 3 (BE3) gene-editing tools, the team reduced cholesterol levels in healthy mice treated in utero by targeting a gene that regulates those levels. Also used prenatal gene editing to improve liver function and prevent neonatal death in a subgroup of mice that had been engineered with a mutation causing the lethal liver disease hereditary tyrosinemia type 1 (HT1).