Tagged with Rare diseases

Korea Korean Pharma company, Hanmi has won orphan drug status this week from the US Food and Drug Administration (FDA) for HM43239, a drug for the treatment of acute myeloid leukaemia (AML).   The endorsement of FDA’s Orphan Drug Designation (ODD) — invented to smooth the development of therapies for life-threatening…

Innovation Patient groups oftentimes serve as the authority on a specific disease when it comes to advancing research.   “Without patient perspective, there is no good medical product development,” Rachel Sherman, the FDA’s principal deputy commissioner proclaimed during her keynote at last week’s NORD’s Rare Disease & Orphan Products Breakthrough Summit.…

Mexico David Peña Castillo is the President and founder of The Mexican Federation of Rare Diseases (FEMEXER). Here he discusses what makes a patients association. When we find a way to communicate the complexities of rare diseases simply, we can avoid isolating anyone and the potential to uncover important information is…

USA After her son was diagnosed with SYNGAP1 – an extremely rare gene mutation – Monica founded Bridge the Gap – SYNGAP Education and Research Foundation to support families of those suffering from the illness and to accelerate the path to better therapies. Here she discusses the importance of community for tackling rare…

France Genethon, a laboratory created by the patient organization AFM-Telethon, is one of the world’s leaders in the development of biotherapies for rare diseases. In this interview Genethon’s CEO Frédéric Revah explains its role in the discovery of innovative therapies since the 1990s and the recent launch of YposKesi, its innovative…

Turkey Two months into the role, Umut Meriç, general manager for Chiesi Turkey, discusses the affiliate’s rapid progression and his plans to ensure Turkish patients benefit from the global company’s latest therapies in the respiratory and rare diseases areas. Meriç underlines the importance of motivating and developing a productive, dynamic and…

UK Offering genomic sequencing and analysis, Congenica operates in the exciting world of genomic diagnosis, with a particular focus on rare diseases. Its CEO David Atkins describes the potential of Congenica’s platform and talks commercialisation. Can you please start by introducing Congenica to our international readers? “As well as disease diagnosis,…

UK Sean Richardson discusses Alexion’s focus on rare and ultra-rare diseases, the level of support for these therapies from the UK government, awareness raising, diagnostics, and his future priorities.     Let’s please start this interview by introducing the UK affiliate of Alexion to our audience. How would you describe the…

Hong Kong K P Tsang, president of the Hong Kong Alliance for Rare Diseases (HKARD), shares the challenges faced by patients with rare diseases and their caregivers in Hong Kong, the areas of improvement when it comes to rare disease policy, the advocacy work that HKARD is undertaking, and his insights on…

UK Three years into his role as general manager of Sobi’s UK and Republic of Ireland operations, Neil Dugdale introduces the scope of operations of the Swedish rare disease player to our audience, highlighting the company’s unique positioning in working closely with patient groups to drive forward better understanding of unmet…

Spain Alba Ancochea of FEDER, the association that groups together Spain’s 337 rare disease patient organizations, explains how it interacts with both government and industry to the benefit of rare disease patients, the evolving situation for these patients in Spain, and her strategy for the future. Could you please start by…

Portugal Alnylam, pioneers in the RNA interference field, seek to bring their revolutionary medicine across Europe, and in particular Portugal before 2020. In this interview the new General Manager for Spain and Portugal, Alicia Folgueira Lopez, a rare diseases authority, discusses how to build an affiliate from scratch, the synergies across…