31th July 2019: AcceleRare partners Prof. Gerald Schwank (ETH Zürich) and Prof. Johannes Häberle (Children’s Hospital Zürich) recently applied a novel gene editing tool to correct mutations that cause the metabolic disease Phenylketonuria.  Using viral vectors to deliver the editing enzyme to the liver, they reached repair rates up to 60%, and fully reverted the phenotype of the mouse model. Their work has been published in the journal Nature Medicine. Access the article here.

25th July 2019: Funding for rare disease translation at an AcceleRare partner institution
Gain Therapeutics SA Announces Award Notification of a €1.4M Grant Support from Eurostars-2 together with the Institute for Research in Biomedicine and Neuro-Sys SAS:

18th July 2019: The end to the diagnostic odyssey
A recent report made by the Rare Diseases UK highlighted the problem of a striking lag between occurring symptoms and proper diagnosis of patients with rare diseases. 
According to the report, a diagnosis can take on average 6 years during which 8 doctors are engaged in the diagnosis process. What is more, in some cases, the diagnosis remains elusive even for decades.
As you can read in the attached piece (link), knowledge about the molecular basis of an orphan disorder can catalyse chain of events that would affect the fate of the patients, their families, healthcare professionals and also researchers around the world.
Rare Diseases UK is pushing the field forward in order to find successful treatments and cures for rare diseases.

01st July 2019: Research on rare diseases often yields important information on genes and pathways operating under healthy conditions, as well as in common diseases. This concept is at the core of an extensive review by Olivier Devuyst and colleagues, just published in the prestigious journal Physiological Reviews (van der Wijst J et al.Physiol Rev 2019; 99: 1575-1653).

30th June 2019 On Sunday 21st of July 2019, the Forderverein fur Kinder mit Seltenen Krankheiten (KMSK) organizes the KMSK Zirkus Erlebnis. On this day, healthy siblings (aged 6-16 years old) of children with rare diseases will prepare and perform a show for their siblings, parents, and grandparents. Joy for all guaranteed! More information and registration via the events section of the KMSK website.

The KMSK supports children with rare diseases and their families.

27th June 2019 Last week, we announced that AcceleRare has been shortlisted by the Swiss National Science Foundation (SNSF) after a thorough 1,5-year selection procedure for the 5th round for Swiss National Centers of Competence in Research. This means that AcceleRare fulfills all required academic criteria, addresses an urgent societal need, and shows the potential for great scientific breakthroughs. During the coming months, the SNSF will visit the host institutes (for AcceleRare that is the University of Zurich, with ETH Zurich and University of Geneva as co-leading hosts). After a final assessment by the State Secretariat for Education, Research and Innovation (SERI), the final decision will be made in Autumn 2019 by the Federal Department of Economic Affairs, Education and Research.

During this final stage, we need your support! Follow us via LinkedIn and Twitter, and spread the word about AcceleRare. Together, we can advance drug discovery for rare diseases!

24th June 2019 The research group of AcceleRare partner Prof. Dr. Jean-Christophe Leroux published an article on nanoparticle-delivery of nucleic acids. Such safe delivery of DNA has been a major bottleneck for gene therapy. With AcceleRare, we strive to further develop such state-of-the-art methods and bring them rapidly towards the clinic for the treatment of rare diseases.

19th June 2019 AcceleRare is still in the running to become a National Center of Competence in Research. After further assessments by external experts and an interview at the Swiss National Science Foundation, we have made it to the next stage in the selection procedure. The AcceleRare team is thankful for the positive response to our research program, and we are further strengthened in our belief that the time has come to structurally advance drug discovery for rare diseases. Want to know more and get involved? Contact us for more information!

7th June 2019 Drug discovery is a multidisciplinary effort that involves experts from diverse scientific fields, which need to communicate clearly with each other. Effective knowledge creation requires both formal and informal organisational structures and practices. With Professor Georg von Krogh and his team from ETH Zurich, AcceleRare will investigate and address strategic management aspects to improve academic drug discovery for rare diseases. Read more about their work in a recent paper about drug discovery in pharmaceutical industry.

1st of March 2019 Today it is international rare disease day, with which we would like to raise awareness for rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families. AcceleRare collaborates with ProRaris, the Swiss alliance for rare diseases, which will host several events.

ProRaris, the Swiss alliance for rare diseases

22nd February 2019 The Swiss Federal Office of Public Health published an overview of projects and measures that have been undertaken since the publication of the National Rare Disease Policy in 2014. Although some progress has been made, there remains a lot to do. With AcceleRare, we aim for structural solutions in the field of rare disease research.

24th January 2019 The 7th RADIZ Rare Diseases Summer School will be be held from 10-12 July 2019 in Kartause Ittingen, Warth (Switzerland). Through lectures, workshops, poster sessions, and oral presentation, it will address a wide variety of subjects related to rare diseases, from disease mechanisms and animal models, to improving diagnoses, to novel therapeutics. More information and registration here.

23rd November 2018 AcceleRare Partner Prof. Dr. Matthias Baumgartner from the Kinderspital Zurich investigates rare metabolic disorders. His work on Andrin’s disease was featured in the UZH news. Read the article.

10th October 2018 The Drug Discovery Network Zurich (DDNZ) organizes a symposium on ‘Academic Drug Discovery for Rare Diseases’. The program focuses on how cutting-edge technologies in basic research can be exploited to serve unmet medical needs of rare disease patients. More information and registration via the DDNZ website.

Program for the 2018 DDNZ Symposium ‘Academic Drug Discovery for Rare Diseases’

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