The providing  and editing of the national rare diseases document is one of the most vital activities of the Rare Diseases Foundation, and in this regard, the Rare Diseases Foundation of Iran, with a lot of endeavor  and follow-up, and with the participation and cooperation of the professors and expertise of the Foundation and other universities of medical sciences, prepared and compiled the National Document of Rare Diseases of Iran, which after being approved by the Secretariat of the Supreme Council of Health and Food Safety and the cooperation and collaboration of the honorable officials of that ministry and other related institutions, was finally approved by the honorable president and announced. Undoubtedly, the declaration of this document can be a very huge and effective step in determining the duties of the institutions and beneficiaries related to these patients, providing services to rare patients and their families, and solving the problems of these patient respectfully

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The deputy of insurance and services of the iran health insurance organization declared :we cover one million and  thousand patients in the fund for rare  and hard treatment diseases.

In report of mehr news agency ,Mehdi rezaei,

According the insurance coverage of rare and hard treatment patients, announce : 1,600 billion tomans have been paid in the first 4 months of this year to cover the costs of rare and hard treatment patients .

Most of services are provided in the pharmaceutical, medical radiation and laboratory services, 75% of which are related to the pharmaceutical sector .

Rezaei added: 63 service packages have been prepared in this fund for these patients, who have received the most services in the rare and hard treatment patients fund, respectively , for cancer , MS, AND HEMOPHILIA

https://www.mehrnews.com/news/5862708

Neurofibromatosis type 1 (NF-1) is a Neurocutaneous Syndrome with autosomal dominant mode of inheritance and has a high propensity to develop benign and malignant nervous system tumors.

Advances in genetic understanding of gorlin syndrome and emerging treatment options

 Shawn Shih, Christina Dai, Ahmed Ansari, Brittany A Urso, Amy I Laughlin & James A Solomon

Introduction: Basal cell carcinoma nevus syndrome (BCCNS) is a rare syndrome characterized by multiple basal cell carcinomas (BCC), odontogenic keratocysts, and other abnormalities. The most common etiology, the loss-of-function PTCH1 mutations and consequent constitutive hedgehog signaling, can be blocked by smoothened inhibitors (SIs). However, other causes and alternative pathways have been identified.

A General Strategic Review on Rare Diseases & Orphan Drugs

Joan Chambers

Senior Strategic Advisor      

Rare diseases are generally defined as diseases that affect fewer than 200,000 people in the United States. Because rare diseases touch so few people, pharmaceutical companies traditionally did not pursue drug treatment for them. In addition, only a few types of rare diseases are tracked in the United States. That makes it more difficult to know exactly how many rare diseases exist and how many people have them.¹ Treating these diseases has its own unique challenges, said Scott Schliebner, vice president of scientific affairs – rare diseases for PRA Health Sciences. Schliebner, considered an industry expert on this topic, is also a well-recognized speaker about rare diseases.