China After a Big Pharma career in the US, Dr Yao-Chang Xu returned to China and in 2016 founded Abbisko, a biotech that began with a focus on small molecule oncology treatments. He shares the company’s evolution over the past eight years, including its exploration of broader therapeutic areas, the major…
USA The Promising Pathways Act 2.0 is a newly proposed bill that aims to expedite and streamline approval pathways for rare disease therapies in the US. The Act is, however, facing a number of challenges in terms of implementation. The sponsors of these niche therapies tend to be small and medium-sized…
Saudi Arabia Mohamed Abu Shawish of Japanese rare disease specialist Kyowa Kirin outlines how, by focusing on initiatives like genetic testing programs and facilitating treatment delivery to remote areas, the company ensures that rare disease patients in Saudi Arabia receive timely diagnoses and access to essential treatments. He also explains how the…
Saudi Arabia Karim Smaira and Kamel Ghammachi, founders of Genpharm, underscores the strategic significance of the recent majority acquisition by Abdul Latif Jameel Health, part of Abdul Latif Jameel, driven by a shared vision and the imperative for accelerated growth. They discuss their expansion into diverse therapeutic areas, emphasizing the importance of…
Portugal Diabetes is a rising threat to the Portuguese people, the country’s healthcare system, and its economy. Portugal’s National Diabetes Observatory observed that 1.1 million Portuguese people between the ages of 20 and 79.5 (out of a total population of just 10.3 million) were living with diabetes in 2021. This…
Europe A new piece in the January 2023 edition of DIA’s Global Forum magazine lays out how a new coalition aims to transform the landscape for rare disease treatments in Europe and beyond. The knowledge and infrastructure required to unlock rare disease “white spots,” diseases where there is currently no…
USA Drawing on her experience as rare disease patient advocacy group SYNGAP1 Foundation’s founder and former president, Monica Weldon argues for the need to standardize genomic testing practices, warns against the possible ethical implications of individualized medicine, and makes a cause for a more holistic approach within rare disease communities. …
UAE From humble origins, taiba can now count on a unique two decades of experience in bringing innovative rare disease products to patients across the MENA region. The Dubai headquartered company, now branding itself as the partner of choice for rare diseases across the region, has also moved to create a…
UAE Erstwhile general manager for the GCC countries at Japanese-headquartered rare disease specialist Kyowa Kirin, earlier this year Myriam Hakim was appointed as the firm’s regional nephrology franchise head for the entire EMEA region. Here, Hakim discusses some of the key challenges she navigated the GCC grouping through over the past…
UAE Ahmad Abu Dahab became the first Middle East employee for Swedish-headquartered haematology, immunology and specialty care player Sobi back in 2012 and now serves as VP for the entire Middle East, Turkey, and Northern Africa region. Abu Dahab explains some of the key trends at play in the rare diseases…
UAE CSL Behring – which specalises in plasma-derived, and recombination therapeutic products – has undergone significant expansion in the Middle East and Africa (MEA) region in recent years under Hassan Herrou. He explains why building partnerships with the relevant authorities as well as patient associations is vital to fostering access in…
UAE Mohamed Abu Shawish discusses some of the core challenges facing rare disease patients in the GCC region and how Kyowa Kirin is engaging in multi-stakeholder partnerships across the healthcare sector to help solve them. It is important that if a patient is diagnosed, the treatment is always readily available,…
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