Tagged with Rare Disease

France Franck Puget, managing director for CSL Behring in France, discusses the blood plasma specialist’s commitment to France, building bridges with public authorities, and regulatory attitudes towards rare disease treatments.   Our neighbours envy the Temporary Authorization of Use (Autorisation Temporaire d’Utilisation / ATU) system that we have in France, and…

Opinion Our contributor at large, David Crean, Managing Director for Objective Capital Partners, attended J.P. Morgan’s 2019 Health Care Investor Conference, one of the biggest events in the life sciences calendar and a fantastic indicator of what is to come in healthcare for the year ahead. Here, Crean gives us the inside scoop. …

Opinion 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 Monica discusses the incredibly low rate of treatment options for…

Spain Giuseppe Chiericatti of Chiesi Spain talks strategy, the development of the affiliate and shifting attitudes towards rare diseases in Spain.   Social responsibility and sustainability is a key part of our mission. Our goal is to integrate the concept of sustainability in whatever we do You’ve been leading the Spanish…

USA PJ Brooks PhD, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA, highlights the importance of going beyond one disease at a time in clinical trials, some of the roadblocks that still remain…

Europe 125 medicines for rare diseases were approved by the EMA between 2004 and 2014, of which 71 were granted orphan drug status. This chart shows the availability of 112 of these drugs across European countries. The largest number of medicines for rare diseases were in continuous use in Germany and…

Turkey Levent Canyurt, CEO and founder of BIEM Ilac, shares how he transformed the company from a domestic pharmaceutical distributor into an international player, with an innovative portfolio for the treatment of rare diseases. He also highlights the importance of greater collaboration between stakeholders in the Turkish biotech industry and gives…

Turkey Abidin Gulmus, founder and chairman of Gen Ilaç, describes the company’s impressive journey to become Turkey’s principle orphan drug distributor and shares his vision for leveraging the company’s domestic expertise to expand their reach into foreign markets.   Gen Ilaç is one of the fastest growing Turkish success stories although…

Malaysia Sea cucumbers – odd-looking marine animals with leathery skins and elongated bodies – can be found on ocean floors worldwide. Although more famous as a culinary delicacy in East and Southeast Asia, one Malaysian company – Healwell – is using these primitive creatures to save lives.   Dr Hassan Yaakob,…

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…