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).

The researchers were able to trace the origin of CAID by using Quebec’s BALSAC genealogical database. Their goal was to predict in which parts of the province the disease is most likely to appear in future generations, depending on the areas where there are the greatest numbers of carriers of the recessive gene that causes the disease. They believe this information may help the medical system save money on CAID testing in future.

In a study at seven hospitals across the UK and the USA, researchers found the brain scan, called magnetic resonance (MR) spectroscopy. Brain damage affects around one in 300 births in the UK, and is usually caused by oxygen deprivation.

The 7th Scientific Conference of Hypothesis Diseases, Growth Hormone Disorder in children and Adults was held on 21 December 2018 by the scientific secretary, Dr. Mohammad Khamseh and the executive secretory, Dr. Mojtaba Malek from Endocrine and Metabolism Institute of Iran Medical Science University at Parsian Evin hotel, Kooh-e-Noor hall.

Since it can take 5 to 7 years for a patient with a rare disease to receive an accurate diagnosis—which can be the gateway to appropriate treatments and access to life-saving clinical trials—a panel at this year’s National Organization of Rare Diseases’ Rare Diseases & Orphan Products Breakthrough Summit (NORD Summit) held in Washington, DC, delved into specific associated challenges and how providers might overcome them.

Researchers from The Rockefeller University recently uncovered the molecular mechanism which causes individuals with a rare skin condition to be susceptible to human papillomavirus (HPV).

Rare Diseases Foundation of Iran as the First Non-governmental Organization Could Join ICOSEP (International Coalition of Organizations Supporting Endocrine Patients)