Scientists at Scripps Research have found an important immune system-regulating protein in principle could be targeted to treat cancers and chronic viral infections.
The scientists, in a study published November 12 in Nature Chemical Biology, set out to determine the function of a protein, ABHD12, whose absence causes a rare genetic disease featuring a host of brain and nerve problems.
ECRD 2018 took place on 10-12 May 2018, at Messe Wien Congress Center, Vienna, Austria.
“Rare Diseases 360° – collaborative strategies to leave no-one behind” was the overarching theme of ECRD 2018. This theme reinforces the unique quality of this foremost event for the rare disease community in Europe, bringing together and facilitating effective policy discussions between all rare disease stakeholders. It also encompasses the comprehensive range of topic areas covered at the conference and the viewpoint of rare disease patients as equal experts, representing all rare diseases, across borders.
“Is your Child growing normally? Find out! Because a child’s growth is a major sign of their health.”
ICOSEP ( International Coalition of Organizations Supporting Endocrine Patients) _ For decades there has been battle over misunderstanding that children’s growth is a cosmetic. It has been very frustrating as ICOSEP have helped to identify children with brain tumors, adrenal issues and much more who were failing and growth was a major symptom which was discounted. They have made too much effort to raise the public awareness. The only reference resource book correlates to growth is compiled and is free to download: Click Here
Iran signed the UHC2030 Global Compact and we warmly welcome them to the partnership.
The experience of Iran over past decades and most recently with the Health Transformation Plan (HTP) shows the importance of developing and maintaining health systems with a strong foundation in primary health care and local participatory processes to ensure that health needs are met in an affordable way. The recently-launched National Health Assembly demonstrates the unique role of civil society and community representatives in mobilizing political commitment for universal health coverage (UHC), providing a viable link from local-level to national participation and decision-making.
EURORDIS – List of the latest marketing authorisations and orphan medicinal products designations
Detailed information on European orphan medicinal products designation applications is available on the EMA website. A full list of designated and authorized orphan medicinal products in Europe available at: ec.europa.eu.
The table is available here:http://radoir.org/en/wp-content/uploads/2018/07/Recent-Marketing-Authorizations.pdf
Update on the European Commission/EMA–FDA bilateral of 18-19 June 2018
Senior officials from the European Commission (EC), the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) held their 2018 bilateral meeting in Brussels, Belgium, on 18 and 19 June. The two-day bilateral regulatory dialogue allowed the strategic partners to review their ongoing cooperative initiatives, discuss strategic priorities for the coming years and further strengthen the continuous close collaboration with specific action in the field of pharmaceuticals. The collaboration between EMA and FDA formally started in 2003 and has gone from strength to strength. The two agencies have daily interactions, most of them structured around working groups or ‘clusters’, with the aim to advance scientific and regulatory excellence worldwide.
EURORDIS Press- Paloma Tejada, Director, Rare Diseases International (RDI), delivered an official statement at the World Health Assembly of the World Health Organization (WHO) in Geneva at the end of May. The statement is the product of a collaboration between a number of organisations holding the status of ‘special relations with the WHO’ (Thalassaemia International Federation, World Federation of Hemophilia, International Alliance of Patients’ Organizations and March of Dimes) and the umbrella organisations that are members of the NGO Committee for Rare Diseases, including EURORDIS, Ågrenska, the International Alliance of Women, the International Federation for Spina Bifida and Hydrocephalus, and RDI. The statement called on WHO Member States to “not leave behind significant but often neglected rare diseases” and to promote national rare disease strategies, advocate for available and affordable medicines, and develop synergies across borders to ensure access to timely, appropriate and effective diagnosis and treatment.
EURORDIS News- Rare Diseases Europe has been formed in order to help people living with a rare disease and concentrates on the social challenges faced by rare patients like : The gaps in coordination between medical-social and support services .
As the first personalized cell and gene therapies are approved from small clinical trials, researchers propose the creation of publicly accessible databases to pull together real-world results. www.the-scientist.com
Due to advances in rare-disease research and individualized cell and gene therapies, there has been a recent crop of treatments approved by regulatory agencies not based on the “gold standard” randomized, controlled, Phase 3 clinical trial. Rather, drug companies are getting products on the market from small, Phase 2 trials with a single arm—those that treat all patients with the experimental therapy rather than having some on a placebo or standard-of-care treatment.