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Burjeel launches new project to identify rare diseases in Abu Dhabi

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Abu Dhabi: With the support of the Abu Dhabi Ministry of Health, Burjeel Holdings, in partnership with US biopharmaceutical company BridgeBio Pharma, has launched the NADER project, which aims to identify rare diseases in the MENA region and provide patients with leading healthcare options. Clinical trials of the NADER project will be conducted in Abu Dhabi.

The two entities signed an initial, non-binding collaboration agreement establishing a mutual intent to collaborate on the NADER (Needs Assessment and Therapeutic Development for Rare Diseases – “nader” means “rare” in Arabic) program . These diseases are of genetic origin, are usually life-threatening or chronically debilitating, affect a small number of people, and continue throughout a person’s life, even if symptoms do not appear immediately.

The NADER project aims to revolutionize the field of early diagnosis and treatment to improve patient outcomes. Entities intend to collaborate to identify patients with multiple rare diseases including achondroplasia, achondroplasia, FGFR-driven craniosynostosis, autosomal dominant hypocalcemia type 1, limb-girdle muscular dystrophy Type 2I, congenital adrenal hyperplasia, Canavan’s disease, propionate academia, methylmalonate academia, pantothenate kinase-associated neurodegenerative disease, and other diseases by mutual agreement.

Although these rare diseases affect a small proportion of the population and may present with nonspecific signs and symptoms, they are often misdiagnosed or only diagnosed later in the course of the disease after serious complications develop. Accurate and early diagnosis is critical to initiate intervention in a timely manner and avoid disease progression. The project will also allow the identification of patients who have already been diagnosed but currently do not have access to cutting-edge therapies or clinical trial options.

In the first phase of the NADER project, the two entities intend to leverage innovative risk assessment algorithms that will be deployed from secure internal data from healthcare providers to identify patients at risk for specific rare diseases. Expectations for future phases of the project include analyzing large volumes of data to identify disease-associated patterns and markers and generating personalized risk scores for patients, followed by genetic testing to confirm the diagnosis in identified potential patients.

The project also aims to develop awareness and education campaigns about these rare diseases to further support mapping activities and encourage local collaborations with other healthcare providers. Burjeel Holding will deploy the program in hospitals and medical centers across the UAE as part of routine diagnostic care and plans to expand the program through its growing healthcare infrastructure in the region. Local partnerships will also be sought to facilitate wider implementation.

Dr. Asma Ibrahim Al Mannaei, Executive Director, Research and Innovation Center, Abu Dhabi Department of Health (DoH), said: “As part of our ongoing efforts to advance early diagnosis and treatment, Abu Dhabi Department of Health is honored to A leading partnership for the health and well-being of community members.

Building on Abu Dhabi’s position as a leading destination for life sciences and innovation, we remain committed to providing the emirate’s healthcare providers with the means to lead research and conduct clinical trials, charting a path towards discoveries and breakthroughs in the emirate and beyond The road to sexual health care. Initiatives like these exemplify the department’s vision to ensure the health of all while accelerating healthcare outcomes for the benefit of global society. “

John Sunil, Chief Executive Officer of Burjeel Holdings, said: “The NADER project marks a major milestone in our commitment to providing the best possible care for patients. It brings together Burjeel’s expertise in healthcare delivery and promotion knowledge and BridgeBio’s deep expertise in delivering breakthrough therapies for rare diseases. By combining our strengths, we seek to address the challenges of identifying rare diseases, which are often not diagnosed before symptoms become severe. The project will Enables early intervention, whether with existing or new therapies.”

The goal of the first phase is to develop a roadmap to bring new treatment options to identified patients through clinical trials.

Dr. Khaled Musallam, Group Chief Research Officer and Project Leader, Burjeel Holdings, said: “Due to our unique gene pool, many rare diseases are highly concentrated in our region. These diseases are often lost in the system or have very limited treatment options. The ultimate goal of the NADER project It is clinical trials of small molecule and gene therapies that connect patients to evolving treatment options. The first step in bringing such innovative solutions to our region is identifying the disease burden and unmet patient needs.”

Dr. Neil Kumar, Founder and CEO of BridgeBio, said: “Building on our established foundation of care for patients with genetic diseases, we hope that BridgeBio can help more patients gain access to new treatments, clinical trials and capabilities, as well as strong technical support. ” Burjeel, so we are excited to work with them to expand our efforts in the MENA region. BridgeBio’s vision is to serve patients around the world, and this is an important step in that direction. “

As the collaboration unfolds, both Burjeel Holdings and BridgeBio anticipate significant advances in the region’s rare disease arena. The project could also provide patients in the region greater access to state-of-the-art pharmacological and gene therapies that BridgeBio is currently developing.



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