The development of rare diseases therapies requires the right regulatory, economic and political ecosystem to ensure that investments are made in areas where research would not otherwise be carried out.
This symposium, held from the 5th – 9th of February 2018, is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians [...]
Rare Disease Day takes place on the last day of February each year. It is an international event, which aims to raise awareness amongst the general public and decision-makers.
The Portuguese Association for CDG (APCDG) are attending the USA CDG Family Conference. They will be giving a talk on their international research network CDG & Allies - Professionals and Patient Associations International Network (CDG & Allies- PPAIN).
The 1st MetabERN meeting with Patient and Family Associations, which will be held at the Frankfurt Lindner Hotel, Germany, on 13th January 2018. The opinion of patient and family organisations is highly valued by the MetabERN as to build the foundation for developing long-term activities and programmes centred around high-quality patient care. The meeting represents […]
Date: 10-12 May, 2018 Venue: Vienna, Austria The European Conference on Rare Diseases 2018 (https://www.rare-diseases.eu) Co-organised by EURORDIS (www.eurordis.org), DIA, and for the first time Orphanet, the theme of this edition of the conference will be “Rare Diseases 360°: Collaborative Strategies to leave no-one behind”. Importantly European Reference Networks will be a transversal topic running through […]
Date: 2-4 August, 2018 Venue: San Diego, United States This symposium is an opportunity for the MPS and related diseases community to share and exchange new information, to learn about new breakthroughs in science and medicine, and develop strategies to keep us moving forward. As we look to an exciting future filled with opportunities on […]
We are pleased to announce the Recordati Rare Diseases Academy Course entitled Inherited disorders of metabolism affecting the liver: management and outcome. The course is jointly organised by the Hepatology and Metabolic Center, Leuven and the Charles Dent Metabolic Unit, London. It will be held in Leuven Belgium on 8th and 8th March 2018. It […]
The workshop, hosted by Istituto Superiore di Sanita, in Rome, Italy, will focus on the question: How can ERNs generate, appraise and utilise clinical practice guidelines, to enhance the impact and deployment of consensus guidelines. The AIMS of the workshop will be: To agree a list of terminology pertaining to Guidelines, to be used between […]
We wish to announce the Recordati Rare Diseases foundation Focus Course on SYNAPTIC METABOLISM AND BRAIN CIRCUITRIES IN IEM: EXPLORING OLD AND NEW DISORDERs held 16 – 18 November in Barcelona, Spain. The course will focus on the synapse as a highly specialized structure with specific chemical composition and metabolic functions necessary for an appropriate […]