The number of rare diseases reached 432 types.

The third medical commission of Iran’s Rare Diseases Foundation was held on Sunday, November 7, with the confirmation of 10 other rare diseases.

According to the public relations report of the Rare Disease Foundation, these diseases include:

CoQ10 deficiency

Angelman syndrome

Rohhad syndrome (IBS)

Cohen-Gibson syndrome

Cushing’s syndrome

Craniosynostosis

Macrophage activation syndrome (MAS)

Hereditary primary hypomagnesemia

Giant Axonal Neuropathy

Hypophosphatasia (HPP).

This commission was attended by Dr. Yaser Davodian, Chairperson of  Iran Rare Diseases Foundation, Dr. Hamidreza Edraki, CEO of the Foundation and member of the academic faculty of Shahid Beheshti University, Dr. Mehdi Norouzi, Vice President of Research of the Rare Diseases Foundation, Dr. Ali Tagavi, a member of the Scientific Council, and Dr. Mohammad Haqshenas. Internist, Dr. Shadab Salehpour, pediatric endocrinologist and metabolism specialist, Dr. Siamak Abdi, neurologist,
Dr. Hossein Afra, Vice President of Medicine, Dr. Tayebe Khamsi, Vice President of Education, Somaye Hasanpour, Vice President of Rare Diseases, and Roya Tavakoli, responsible for International Affairs of the Foundation, were held.

The US Food and Drug Administration (FDA) has approved zilucoplan (Zilbrysq, UCB) for the treatment of generalized myasthenia gravis (gMG) in adults who are antiacetylcholine receptor (AChR) antibody positive.

Zilucoplan is the first once-daily, subcutaneous, targeted C5 complement inhibitor for gMG for self-administration, the company said in a news release announcing approval.

gMG is a rare, chronic disease that affects almost 200,000 patients in the United States, the European Union, and Japan.

Patients with gMG can suffer a variety of symptoms, including drooping eyelids, double vision, and difficulty swallowing, chewing, and talking.

They can also experience severe life-threatening weakness of the respiratory muscles. In about 80% of patients, the condition progresses to generalized muscle weakness.

Clinically Meaningful Improvement

The FDA approval of zilucoplan for gMG was based on safety and efficacy results from the phase 3 RAISE study, published in The Lancet Neurology in May.

The study randomly allocated (1:1) 86 adult patients who were AChR antibody positive with gMG to receive daily subcutaneous injections of zilucoplan 0.3 mg/kg or placebo for 12 weeks.

“Zilucoplan treatment showed rapid and clinically meaningful improvements in myasthenia gravis-specific efficacy outcomes, had a favorable safety profile, and was well tolerated, with no major safety findings,” the investigators reported.

The most common adverse reactions (≥10%) among patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.

“For people with gMG, the unpredictable nature of the severity and frequency of symptoms can be debilitating and can have a substantial impact on many aspects of their day-to-day lives,” lead investigator James F. Howard, MD, distinguished professor of neuromuscular disease, the University of North Carolina at Chapel Hill School of Medicine, said in the release.

“Zilucoplan demonstrated rapid improvements in gMG symptoms at Week 12, with differences seen as early as one week, and provides a new treatment option for a broad population of AChR antibody-positive gMG patients,” Howard added.

In June, the FDA approved rozanolixizumab (Rystiggo), also from UCB, for the treatment of adults with gMG who are positive for AChR or anti-muscle-specific tyrosine kinase (MuSK) antibody, as reported by Medscape Medical News.

Rozanolixizumab is a subcutaneous-infused humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor, reducing the concentration of pathogenic IgG autoantibodies.

“This is an important development for the community because, with more FDA-approved treatments for generalized myasthenia gravis, physicians have additional tools to treat this disease in individualized ways that are the right fit for each individual patient,” Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America, said in the news release.

http://www.medscape.com

1 * Hamid Reza Edraki and 2 Safora Ghazimorad
1
Neuroradiologist (LMU Munich -Germany), Faculty
member of Shahid Beheshti University of Medical Sciences,
Tehran, Iran.
2
Master’s degree in psychology, a member of the Iranian
Psychological System Organization, a member of the Iranian Neuropsychological Association and APA,

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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 statistics of the nine months of 1401 in the pharmaceutical system of the country show that some manufacturers that produce drugs in our country  has included a number of drugs related to rare diseases in their production inventory. Among them, the following drugs can be mentioned:

1- Anakinra INJECTION, SOLUTION 100mg/0.67 ml (Persisgen)

2- Bevacizumab INJECTION, SOLUTION, CONCENTRATE 25 mg/ml (Aryogen – Acteropharma)

3- Biotin TABLET 5 mg (Iran darou – Aburaihan- jalinous – rahapharm – Oxin)

4- Biotin AMP 5 mg (actover co- darou pakhsh- exir co- pbjpharma- Oxin- Aburaihan- Tadbir kala- alborz)

5- Bortezomib INJECTION, POWDER, LYOPHILIZED 3.5mg (Nano alvand-

Sobhan darou- ‎ACTe-HRJOBBOFFER– Rooyan Darou-  nanocedaL CO)

6- Cladribine INJECTION, SOLUTION 1mg/ml (Nano alvand)

7- Cladribine INJECTION, SOLUTION 2 mg/ml (Nano alvand)

8- Brimonidine Tartrate SOLUTION  DROPS 0.2% (Sina darou- Darou pakhsh)

9- Factor VII VIAL 4.8 mg (Aryogen pharmed)

10- Factor VIIa INJECTION, POWDER, LYOPHILIZED 1.2 mg (Aryogen pharmed)

11- Nitisinone CAPSULE 5 mg (osvepharma)

12- Miglustat CAPSULE 100 mg (Modavapharma- faranshimi)

13- Nitisinone CAPSULE 10 mg (osvepharma)

14- Idarubicin HCl INJECTION, SOLUTION- 1mg/ml(Nano alvand)

15- Pilocarpine Nitrate SOLUTION/ DROPS 0.04 (Sina darou- Bbpharmaco)

16- Pirfenidone TABLET 200 mg (Darou darman arang- Mahban group- Modava pharma)

17- Silver Sulfadiazine CREAM 10 mg/g 50 g (Sobhan darou)

18- Sapropterin DiHCl SOLUBLE TABLET 100 mg (Modava pharma)

19- Silver Sulfadiazine CREAM 1% 50g (Iran najo- Sina darou- Emad darman pars- Behvazan darou- Sobhan darou- Iran avand far)

20- Rifampicin CAPSULE 300 mg (Hakim pharm)

21- Sodium Benzoate TABLET 500 mg (Amin pharma)

22- Trientine DiHCl CAPSULE 300 – 250 mg (Zist darou- nanoproduct)

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

In The Name of God, The Beneficent & The Merciful

Pain and sufferings of a disease will ruin the patient’s patience and the strength of his family unless by receiving sympathy and empathy from the society that never ever leaves him alone and helpless which also makes him leaning on the Omnipotent God for the strength when life is overwhelming, morally and financially.

The 8^th of Esfand (27^th February) officially dominated “National Rare Diseases of Iran” to support the Iranian Rare Patients. I hope the day denominated in the Iranian calendar to be a different day in this year for the rare patient’s communities, calling for public attention, media and the authorities promising the better days for the patients as well as promoting the country’s social capital, potentiality. Patients suffering from the rare diseases feel lonely and anxious much more than the others due to the lack of access to the orphan drugs, the expertise specialists and high cost of pharmaceuticals and the necessary medical equipment for the rare patients’ treatment. Each member of the Iranian society feels the rare patients’ pains and grief deeply in heart as a public health challenge. We will do our full and best in our country to remove their pains and sorrows.

The necessary expertise to treat, having access to the orphan drugs as well as the relevant medical equipment and providing the moral and financial supports for these patients and their families have been prioritized and instructed for each Iranian authority and entity. The Government should promote public awareness raising about the rare diseases and their prevention, education, sign and symptoms, consequences, communicating with the rare patients, meeting their unmet treatment needs for orphan drugs and medical equipment as a significant obligation in taking action. Based upon the approval of “The National Strategic Plan for Rare Diseases in Iran”, it is forcefully assigned to the Ministries of Health & Medical Education, Labor & Social Welfare, Education and the organizations of State Welfare, The Imam Khomeini Relief Foundation and all the relevant organizations and centers. In accordance with the vulnerability of rare patients, their immunization and treatment should be considered of high priority during Pandemic COVID-19 by the Ministry of Health & Medical Education.

In the end, the immense efforts and efficient proceedings by the public groups, medical personnel and all the colleagues at various governmental sector are highly appreciated. I hope to make the national strategic plan for rare diseases in Iran such effectively in near future for the kind attention of the rare patients and people of our country accompanied by the respected Islamic parliament of Iran. My fulfilled wishes reminded of God’s grace and almightiness for all the patients and my sincere appreciation to the compassionate healthcare entities and personnel of the Rare Diseases Foundation of Iran (RADOIR) for their hardworking and immense support of the Iranian rare patients and their families despite of the imposed offensive sanctions.

Wa Salaam-Alaikum

Seyyed Ebrahim Raisi

President of the Islamic Republic of Iran

In a meeting  on Monday October 11, 2021 between the Chairman and Managing Director of the Rare Diseases Foundation of Iran (RAADOIR) with UNICEF resident representative in Iran, it was decided to hold a collaborative working group for the right of the Iranian rare diseases children.

During the first meeting session held at UNICEF head office in Iran, Yaser Davoudian, RADOIR chairman stated that there are 355 types of rare diseases identified at RADOIR’s patient registry system (SABNA) and about 3 million rare patients living in Iran of which the most affected populations are the children suffering under dissatisfactory treatment condition, unfortunately.

He also added,” Due to cruelty of the imposed sanctions, lack of access to treatments and orphan drugs as well as not enough awareness and knowledge among the medical and public societies about such life threatening diseases, the Iranian rare children are intensively suffering.”

Davoudian continued,” The imposed sanctions by the American government are against UN human rights policies led to the Iranian rare children’s right abuse and neglect. Yet we at RADOIR as an independent NGO, have made invaluable achievements like development of National Strategic Plan of Rare Diseases in Iran as the 30^th country in the world as well as identification the rare patients not only in Iran but the Middle East which have encouraged the countries in the region to apply membership at our foundation.”

He emphasized that up to now, we have not received any fund from any local and international organizations and have been able to successfully keep on our mission despite of the existing sanctions and restrictions. RADOIR chairman added,” We have still many action plans for the rare patients which in collaboration with UNICEF and WHO, hoped to reduce the rare diseases children’s pains and sufferings and this is the least to be enacted by UNICEF and other referral organizations in Iran.”

Yaser Davoudian declared,” We expect UNICEF entities in Iran to meet the needs of the Iranian rare children like others all over the world along with the global heath supportive goals through developing a collaborative strategic plan with RADOIR to find the best solutions, accordingly. We do not intend to meddle with financial assistance or diplomatic policies. RADOIR is asking for the efficient suggestions and facilities at UNICEF to manage the support for the vulnerable rare children in Iran.”

Dr. Hamid Reza Edraki, managing director of the Rare Diseases Foundation of Iran who has been awarded by the Iranian president as the honorary radiologist for 2 years stated, “ At our foundation, we have achieved scientific success in discovering novel advanced methods of Fetal MRI early diagnosis in brain disorders in the first trimester of pregnancy to prevent rare diseases in children.” Dr. Edraki emphasized that the collaboration of UNICEF and RADOIR will be a turning point in improving the living quality of the Iranian rare children and appropriately educating their families as well as the society about rare diseases.

Dr. Robin Nandy, UNICEF representative in Iran explained,” There are different departments at UNICEF as of Health, Education and other issues with children. Our vision is far from the political affairs and our mission is to find the best solutions to cover the problems and sufferings of children to access a better life. Our first and main concern at UNICEF is to achieve the children’s rights and meeting their needs wherever in the world. “

Dr. Nandy congratulated RADOIR board of directors for their achievements especially implementing rare patients registry system to identify rare patients and improve means of supporting them.

At the end of the session, UNICEF representative in Iran stated.” There is a bright new horizon for the Rare Diseases Foundation of Iran. We will ask WHO resident representative and the international department of MOH-Iran bodies to join our think tank session with RADOIR in very near future.”

This meeting was held following the interview session by Kamran Najafzadeh, RADOIR ambassador in Manhattan with Dr. Aboubacar Kampo, Director of Health Programs at UNICEF.

Kamran Najafzadeh had an interview with Dr.A. Kampo, UNICEF Director of Health Programs at the United Nations.

RADOIR NEWS- At the interview session with the UNICEF Director of Health Program, the report letter including RADOIR’s achievements as well as orphan drugs needs for rare diseases children treatment by Yaser Davoudian, Chairman at the Rare Diseases Foundation of Iran, was presented by RADOIR ambassador, Kamran Najafzadeh.

RADOIR’s ambassador asked UNICEF director of health program for organizing and prompt a meeting session for the board of directors of Rare Diseases Foundation of Iran with UNICEF resident representative in Iran. During the interview session, Dr. Kampo discussed the subject with Dr. Robin Nandy, UNICEF resident representative in Iran to arrange a meeting appointment with RADOIR in the upcoming week on a phone call.

Dr. Kampo added,” We will follow up the problems with the rare diseases children of Iran with WHO as well and will try to find the solutions, accordingly”. This interview session was arranged by Dr. Majid Takht Ravanchi, Iranian diplomat and the Ambassador of Iran to the United Nations and follow ups by Hamid Taavoli, the Iranian Counselor at Permanent Representative of Iran to the United Nations.