Tagged with Rare Disease

USA Focused on rare and ultra-rare genetic diseases, Ultragenyx identifies itself as the industry’s most productive company working in the field of high un-met need diseases. With no fewer than four products approved across five indications, a blockbuster therapy that reached 2023 revenues of USD 328 million, and six candidates in…

Mexico Nicolas Linares of Ultragenyx Pharmaceutical Mexico highlights the company’s commitment to developing innovative treatments for rare and ultra-rare diseases. He emphasizes the importance of collaboration with healthcare systems and the need for a focused approach to address the unique challenges faced by patients in Mexico.   By fostering collaboration among…

Mexico David López García, a pharma industry executive and rare disease expert, discusses the challenges and advancements in treating rare diseases in Mexico. He highlights the importance of public-private partnerships and the growing interest from industry in the rare disease field.   Every day is an opportunity to make a difference…

China Dr James Xue, founder, chairman and CEO of CANbridge Pharmaceuticals shares the career journey that led him to China’s first rare disease-focused company and the role CANbridge is playing in shaping policies relating to rare diseases, such as the National Rare Disease List. Xue also outlines the company’s robust pipeline…

Mexico Dr Jesus Navarro explains how his personal experience with his daughter’s rare disease led him to form the Mexican Organization for Rare Diseases (OMER) to support others facing similar challenges. Dr Navarro highlights the lack of a comprehensive registry for rare diseases in Mexico and the need for specific legislation…

China General manager Guillaume Delmotte explores Ipsen’s strategic initiatives in China, highlighting the importance of innovation, addressing healthcare challenges, and fostering a sustainable, people-centric organization. Delmotte emphasizes the rapid pace of change and the vital role of human connections in driving success.   Moving [our China headquarters] to Shanghai allowed Ipsen…

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…

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.  …