The MENA Organization for Rare Diseases is collaborating with Burjeel Holdings on the groundbreaking initiative known as NADER (Needs Assessment and therapeutics Development for Rare diseases) to expand the footprint of the initiative across the MENA region. This innovative project is expected to revolutionize the field of early diagnosis and treatment of rare diseases in the UAE and the wider MENA region.

NADER, a project that draws its name from the Arabic word for “rare,” aims to create a paradigm shift in how rare diseases are diagnosed and treated. Recently, Burjeel Holdings, one of the largest healthcare service providers in the MENA region, and BridgeBio Pharma, a leading US-based biopharmaceutical company with a focus on rare genetic diseases and cancers, have signed an agreement to launch operations from Abu Dhabi to conduct clinical trials and research.

Supported by the Department of Health, Abu Dhabi, NADER will leverage the advanced innovation infrastructure and life sciences capabilities of the Emirate to initiate its operations.

MENA Organization for Rare Diseases, with its established network and large experience, will play a pivotal role in expanding patient identification programs across the MENA region, facilitating research initiatives, and promoting clinical trials. The organization will forge strategic partnerships with key opinion leaders, healthcare institutions, and local societies focused on rare diseases to ensure the success of this transformative initiative.

“We are thrilled to collaborate with Burjeel Holdings on the NADER project. This strategic alliance marks a significant milestone for the rare disease community in the MENA region. Together, we can amplify our efforts to enhance patient care and ensure that no one is left behind when it comes to accessing appropriate diagnosis and treatment.” said Prof. Ayman El-Hattab, Founder and President of MENA Organization for Rare Diseases.

“At Burjeel Holdings, we recognize the challenges faced by individuals with rare diseases and their families, especially when it comes to availability of treatment options. With this new collaboration, we will be able to better understand the unmet needs for patients across the MENA region so that we drive the project towards addressing those specific needs,” Dr. Khaled Musallam, Group Chief Research Officer of Burjeel Holdings and project lead.

Project NADER will enable identifying patients with several rare diseases, including achondroplasia, hypochondroplasia, FGFR-driven craniosynostoses, autosomal dominant hypocalcaemia type 1, limb girdle muscular dystrophy type 2i, congenital adrenal hyperplasia, Canavan disease, propionic acidemia, methylmalonic acidemia, pantothenate kinase associated neurodegeneration, and others. 

NADER’s first phase entails the identification of patients using state-of-the-art risk assessment algorithms, identification of patterns and markers associated with evaluated diseases, genetic testing for accurate diagnosis, and public awareness campaigns to educate communities about rare diseases.

The second phase of the project will involve conducting clinical trials for novel therapies to address the urgent need for effective treatments for rare diseases. By merging expertise from both medical and research fields, NADER aims to provide a comprehensive solution to the challenges posed by rare diseases, enhancing patient outcomes and quality of life.

Rare diseases, affecting less than 1 in 2,000 individuals, often present unique challenges due to their scarcity and limited available therapies. Recent data indicates that over 10,000 different rare diseases exist, collectively impacting more than 5% of the population. Yet only 5% of these diseases currently have available treatments, leaving patients and their families with significant unmet needs.

The MENA region, encompassing over 20 countries with a population exceeding 600 million, faces a higher prevalence of rare diseases due to shared cultural factors such as larger family sizes, higher maternal and paternal ages, and elevated rates of consanguinity. The collaboration between MENA Organization for Rare Diseases and Burjeel Holdings signifies a big step forward in addressing this pressing healthcare issue across the region.

About Burjeel Holdings: Founded in 2007, Burjeel Holdings is one of the leading private healthcare services providers in the MENA region. With a network of 62 assets, including 16 hospitals and 24 medical centers, as well as pharmacies and other allied services, the group provides the highest standard of patient care in the region. Burjeel Medical City, the flagship facility of Burjeel Holdings, is a 400-bed multi-specialty hospital and quaternary care center located in Abu Dhabi, UAE. It offers high-quality specialized treatment and complex care in over 40 adult and pediatric specialties, aided by state-of-the-art medical technology and an international team of experts. Find more information at www.burjeelholdings.com.

About MENA Organization for Rare Diseases: MENA Organization for Rare Diseases was established in Dubai advocating for rare diseases in the MENA region. Its goals are to educate, connect, and support. The organization aims at spreading knowledge and increasing awareness about rare diseases among healthcare providers, patients and families, and the public through conferences and meetings, social media, and online newsletters and educational webpages. The organization also works on connecting patients and doctors to exchange experience through establishing support groups for patients and networks for healthcare providers. In addition, the organization supports patients through facilitating receiving services from healthcare institutions and community services. Find more information at www.menararediseases.org.