4 – 5 November, Dubai, UAE

with attendance of  Dr yaser davoudian

The vibrant city of Dubai hosted the highly anticipated Asia-Pacific Regional Meeting of the World Patient Alliance (WPA) on November 4-5, 2023. The gathering brought together global leaders from patient organizations, passionate patient advocates, and dedicated healthcare stakeholders.

A dynamic combination of face-to-face and virtual interaction: One of the outstanding features of the Asia-Pacific Regional Meeting was its hybrid format. This forward-thinking approach allowed participants to connect both in person and virtually, ensuring a diverse and inclusive experience that transcended geographic boundaries. This reflects WPA’s commitment to creating meaningful engagement for a global audience.

Insights from keynote speakers: The event featured a mix of keynote speakers from national and international health organizations, each bringing their unique perspectives to the fore. Insights shared by Mondher Letaief, Shin Ushiro, Lamya Alhazani, Candace Henley, Christiane El Ferzli, Nicole Sheahan, Kimberly Galia, and in-house speakers Andrew Spiegel Esq., Penney Cowan, and Hussain Jafri added depth to the discussions. Overview of key topics: The diverse range of topics covered at the Asia Pacific Regional Meeting provides a comprehensive look at the challenges and opportunities in patient advocacy. From “The Art of Patient Advocacy” to discussions on “Universal Health Coverage” and surveying “Health Emergencies in the Region”, each session contributed to a comprehensive understanding of the current healthcare landscape.

Panel Discussions: Expert Conversation: The event agenda was enriched with panel discussions featuring healthcare experts and patient safety champions. Anas Nofil, Ken Tanda, Edmond Love, Arij Saqer, JS Arora, Mekia Majrashi, Inge Damanti and Regina Kamuga shared their experiences and perspectives, fueling a rich conversation about the future of patient safety and healthcare. Leadership Insights: WPA leadership, including Andrew Spiegel, Penny Cowan, and Hussain Jafari, shared their insights on the success of the event. Their insights highlighted the growing role of patient advocacy in a changing world and expressed appreciation for the global community’s commitment to patient-centered solutions.

A celebration of collaboration and shared dedication: More than just a meeting, the Asia-Pacific Regional Gathering was a celebration of collaboration, dialogue and a shared commitment to improving healthcare for patients worldwide. It provided a fertile ground for making connections, sharing best practices, and exploring solutions that resonate across borders.

The event aims to foster a global dialogue on patient safety, quality healthcare and advocacy. We believe that our active participation will also contribute greatly to your own understanding of the critical issues surrounding patient engagement in healthcare.

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,

---

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

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

---

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)

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