FDA approved “Fedratinib” (Inrebic) capsules for rare adult patients with special forms of Myelofibrosis.

Trikafta (elexacaftor/ ivacaftir/tezacagtor) as the first triple combined therapy for CF mutation has been approved.

Rare Diseases Europe, an alliance of over 800 rare disease patient organisations, is today urging Member States of the EU to take off the table the proposal of the Presidency of the EU Council to withdraw Article 7 of the current proposal for European cooperation on health technology assessment (HTA).

RADOIR’s scientific committee presented the “Atlas of The Rare Diseases In Iran” entitled with the genetic & congenital diseases and published for the first time to the Minister of Health of Iran

The Iranian Radiology Society recognized Dr. Hamid Reza Edraki, managing director of Rare Diseases Foundation of Iran as the best radiologist in Iran for the year 2019

On Friday  Nov. 8^th, 2019 at the venue, Eram Hotel in Tehran,  Dr. Hamid Reza Edraki, managing director of Rare Diseases Foundation of Iran was awarded the medal for the best radiologist of 2019 by the chairman of The Iranian Society of Radiology (ISR), Dr. Jalal Shokouhi and also the special mention for the same recognition was presented to him by Dr. Rabiee, the Iranian Parliament speaker.

Dr. Edraki was born in Boroujerd province of Iran. He is the associate professor at SB Medical University of Iran. He received his MRI fellowship at LMU in Munich- Germany. During the past years, he has been attending at various national & international medical conferences and presented his outstanding researches on early diagnosis of rate diseases, fetal MRI and brain rare disorders, etc.

His last oral presentation on the 4^th semester of pregnancy via fetal MRI has brought him the best of success. It is to be mentioned that Dr. Edraki has also receive the special medal and mention as the best physician for the year 2016 awarded by Dr. H. Rohani , the Iranian president.

The 1^st volume of “ Atlas of The Rare Diseases in Iran focused on the genetic and congenital diseases and disorders was published to share the knowledge and experiences by RADOIR’s experts and researchers with the world’s counterparts.

At a recent meeting of the Council of the International Society for Neonatal Screening, it was decided that the organization would present a plaque to Dr. Louis I Woolf recognizing his seminal contributions to the treatment of phenylketonuria, which made possible, and required the development of neonatal screening for maximum effectiveness.

Spearheaded by the NORD Rare Cancer Coalition, which is comprised of 24 rare cancer-specific Member Organizations, Rare Cancer Day will be observed on October 1 to highlight the challenges people living with rare cancers face and to unify individuals living with rare cancers for awareness and early diagnosis.

 The head of transplant and treatment center of MOH-Iran announced,” There is possibility for accurate diagnosis of MPS patients population to receive further support from the government.”