In addition to the Orphan Products Grants Program that the Office of Orphan Products Development (OOPD) currently administers, a new grant program was established this year by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS) named the FDA Rare Neurodegenerative Disease Grant Program. This new program will be administered…
The upcoming EJP RD training entitled Biobanking in rare diseases: the Poland experience is now open for registration. The 2-day training will be held on November 28-29th online. The training is dedicated to scientists of biomedical sector who want to learn about biobanking standards in rare disease research.Registration is open until November 10th. Register and…
We are glad to announce the launch of the new MOOC entitled “Introduction to translational research for Rare Diseases” will be available. On this five-week course from the European Joint Programme on Rare Diseases, you’ll explore the therapeutic development process from the unique perspective of rare diseases. You will follow the journey from discovery to…
On October 17th, a new MOOC entitled “Introduction to translational research for Rare Diseases” will be available. On this five-week course from the European Joint Programme on Rare Diseases, you’ll explore the therapeutic development process from the unique perspective of rare diseases. You will follow the journey from discovery to approved treatment, learn how therapeutic…
Registration is now open to the yearly 5-day course on “Translational Medicine Explained (TMex) ” on 13-17 February 2023 in Barcelona. The course is intended for PhD/MD students, postdocs and other young scientists who are involved in biomedical research and want to learn more about Translational Medicine. The TMex (“Translational Medicine Explained)” Winter School consists of an introductory e…
In this webinar, that will take place on October 24th from 13:00 to 14:00 CET, Anton Ussi (Operations & Finance Director at EATRIS), together with Agustin Arasanz Duque (Senior Innovation Manager at EATRIS), will provide practical guidance for using two important tools for RD researchers, developed within EU-funded projects: ERICA and EJP RD. This webinar…
EMA is launching a pilot to support the translation of basic research developments into medicines that could make a difference in patients’ lives in the European Economic Area (EEA). The pilot is open to academic sponsors and non-profit organisations who are developing advanced therapy medicinal products (ATMPs). These medicines for human use are based on genes, tissues…
A new article entitled Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases has been published on September 15th. This paper, in which EJP RD members participated in, identifies hurdles in the set-up of six multinational clinical trials for drug repurposing, as use cases. Read…
The World Duchenne Organization is pleased to announce the launch of the Accredited Duchenne Centers Program. This global program is part of the unique development of a worldwide accreditation for Duchenne Centers. It consists of an ongoing development of the process of criteria and visitation leading to the right to be named an Accredited Duchenne…
The registration for the Third International Summit on Human Genome Editing is now open. The event will take place on 6-8 March 2023 at the Francis Crick Institute, London UK. Register for free here The Third International Summit on Human Genome Editing will take place on 6-8 March 2023 at the Francis Crick Institute, London…