December 04, 2020Download pdf
Bresso (Milano); Italy – 4th December, 2020 - EryDel SpA, a global late-stage biotech company aimed at developing and commercializing therapies for the treatment of rare diseases delivered by its proprietary red blood cell technology, announced today its support of the first international campaign raising awareness for Ataxia Telangiectasia.
A-T families from all over the world invite you to pronounce "Ataxia Telangiectasia" (A-T) to raise the visibility of this progressive neurodegenerative rare disease that currently has no therapeutic options and to raise funds for A-T research.
As the families explain in the video (https://youtu.be/JvJ02xwlXmc) “It is difficult to pronounce Ataxia Telangiectasia, but it is more difficult to live with A-T”.
This initiative was launched by the Spanish Aefat A-T patient association and was carried out with the collaboration of nine A-T patient associations from all over the world including Action for AT (UK), Associazione Nazionale A-T (Italy), AT Children’s Project (USA), AT Europe (France), Atileyasam (Turkey), AT Society (UK), BrashAT (Australia), Live Association (Switzerland) and Twan Foundation (Netherlands).
Through this video, families from all over the world evoke the solidarity of all the people who want to join them in supporting the cause by recording a short video in which you pronounce the words "I support research on Ataxia Telangiectasia" and posting it on your social networks in order to shout out the name of AT.
EryDel is eager to contribute in part by spreading the voice “WE SUPPORT RESEARCH ON ATAXIA TELANGIECTASIA!”
About Ataxia Telangiectasia (AT)
Ataxia Telangiectasia is a rare genetic disease caused by biallelic mutations in the ataxia telangiectasia mutated (ATM) gene, for which no established therapy is currently available. ATM encodes a PI3Kinase protein shown to play a pivotal role in response to DNA damage and cell cycle control. Homozygosity for ATM mutations result in a multi - systemic disorder, involving mainly the nervous and immune systems. The major clinical feature of AT is severe progressive neurodegeneration from early infancy. Specific features include progressive ataxia of the trunk and limbs, involuntary movements, oculomotor apraxia, difficulties with speech and swallowing, and delayed peripheral neuropathy. Other clinical features of patients with the classical phenotype include oculocutaneous telangiectasia, immunodeficiency with recurrent respiratory tract infections, radiosensitivity and an increased incidence of cancer.
EryDel SpA is a biotechnology company specialized in the development of drugs delivered through red blood cells (RBCs) by using a proprietary medical device technology. Its most advanced product, EryDex System (EDS) is under late stage development for the treatment of Ataxia Telangiectasia, a rare autosomal recessive disorder for which no established therapy is currently available. EryDex has received Orphan Drug designation for the treatment of AT both from the FDA and the EMA. A completed pilot Phase II trial in AT patients demonstrated statistically significant efficacy of EDS on both the primary and secondary efficacy measures. An international multi - center, Phase III pivotal study, ATTeST, is being conducted. EryDel has a pipeline of preclinical programs that use its proprietary RBC’s delivery technology for the treatment of other rare diseases.
The ATTeST project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667946.
For further information, please contact: Emanuela Germi at +39 02 36504470 or firstname.lastname@example.org