Tagged with Rare Disease

UAE Global advances in rare blood disorders increasingly hinge on where evidence is generated, how patients are followed, and whether innovation reaches beyond a limited few. Khaled Musallam describes how clinical reality at Burjeel informs international research, trial design, and guideline development, while pressing for models that balance scientific ambition with…

MEA CTI EMEA Managing Director Alaa Assem is a physician, ophthalmologist and MBA holder with over 30 years of experience in pharma and clinical research organisations. Since joining CTI, he has strengthened the company’s presence in the Middle East, focusing on rare disease, gene and cell therapy, oncology, and transplantation. CTI…

MEA Rare diseases remain among the most complex and least visible challenges facing healthcare systems, particularly in regions where genetic factors amplify the burden. In this conversation, Ahmed Salem, Head of AstraZeneca Rare Diseases in the Gulf, reflects on how rare disease strategy is translated into regional action, spanning innovation, diagnostics,…

USA Patients and carers with lived experience of a disease have valuable insights to share with physicians and drug developers alike. This is especially true of rare diseases, defined in the US as those that affect fewer than 200,000 people nationwide and which collectively impact over 30 million Americans. For these…

MEA As healthcare systems across the GCC accelerate their focus on innovation, access, and advanced therapies, Novartis is positioning the region at the centre of its long-term strategy. Mohamed Ezz Eldin shares how global priorities are translated into local execution, from rare disease care and genomics to clinical research and innovative…

MEA Biogen’s transformation into a rare disease-focused organisation is reshaping how innovation reaches patients in the GCC. Diederik Kok, the company’s GCC Head reflects on how global strategy translates into regional execution, from launching first-in-class therapies and navigating access for ultra-rare conditions, to building sustainable financing models, clinical research capacity, and…

MEA Hussein Abhari, Head of the MENA region, QIAGEN, has led the company’s regional presence since 2017, spanning products for molecular research and clinical testing. His priorities focus on strengthening long-term partnerships, supporting national healthcare strategies, building local scientific capacity and ensuring advanced molecular and genetic technologies translate into real-world clinical…

USA Chris Peetz, co-founder and CEO of Mirum Pharmaceuticals, reflects on the company’s journey from rescuing overlooked rare disease programs to building a growing global pharmaceutical company. He discusses Mirum’s expanding commercial portfolio, pipeline momentum, and disciplined acquisition strategy, while emphasizing the importance of patient-centered execution in rare disease. Peetz also…

USA Precision medicine increasingly depends on diagnostics that can keep pace with therapeutic innovation while remaining accessible in real-world care. Jonathan Arnold reflects on how QIAGEN approaches oncology and emerging hereditary disease programmes by aligning diagnostic development with pharmaceutical pipelines, prioritising decentralisation, and investing in technologies that support both translational insight…

UAE Dr Rania Alshami reflects on two decades in the Middle East and North Africa clinical research ecosystem, charting the region’s regulatory maturation, rare disease opportunities, and growing role in early-phase trials. She discusses patient access, genomics integration, sponsor engagement, and why MENA is emerging as a credible hub for innovation…

USA As rare disease science accelerates, regulators face growing pressure to adapt without compromising rigour. In this interview, Amy Comstock Rick explains how the FDA Rare Disease Innovation Hub is strengthening cross-centre coordination, evolving evidence expectations for ultra-rare conditions, and creating new spaces for dialogue between regulators, developers, and patients. The…

USA John Hess, Senior Vice President, Americas at Chiesi Global Rare Diseases, reflects on his career journey and the evolution of Chiesi’s dedicated rare disease organisation. In this interview, he discusses the strategic rationale behind establishing Chiesi Global Rare Diseases, the critical role of the US within the global strategy, and…