Dear visitors and patients,

welcome to the homepage of the Section for Translational Neurodegeneration "Albrecht Kossel" of the Department of Neurology at the University Medical Center in Rostock.

The section consists of several research groups, which deal with basic scientific questions about the development and pathophysiology of neurodegenerative diseases (e.g. amyotrophic lateral sclerosis, Niemann-Pick type C, Wilson's disease), as well as with clinical questions and modern concept of patient centered care and the transfer of new findings from basic science into clinical practice (e.g. for improved diagnostics or the therapy of neurodegenerative diseases) and vice versa.

We combine long standing traditions of neuroscientific research at the Rostock University Medical Center by working on neurodegenerative diseases with up-to-date human stem cell-based cell systems. This mainly includes the use of patient-specific models and the development of individualized therapeutical strategies for neurodegenerative diseases.

Thank you for your interest in our work!

Yours sincerely,

Prof. Dr. Dr. Andreas Hermann, Section head

International Parkinson and Movement Disorder Society

Special Series: Approach to patients with genetic choreas (Prof. Emilia Gatto & Dr. Kevin Peikert)

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[Translate to English:] Buchveröffentlichung

We are a supporting partner of the European Rare Disease Network (ERN-RND)

European reference network (ERN):
www.ern-rnd.eu

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News from clinic and basic research

TOFERSEN (Qalsody®) approved for patients with SOD1-ALS in Europe

TOFERSEN (Qalsody®) is an antisense oligonucleotide that binds the so-called messenger RNA of SOD1 (superoxide dismutase 1) and thus significantly reduces the expression of the SOD1 protein. In a pivotal study on SOD1 patients with different mutations in the SOD1 gene, not only was there a reduction in the axonal damage maker neurofilament, but also a slowing of motor decline, measured on the ALS-FRS scale, as well as a prolongation of survival. The drug had therefore been approved by the FDA for the treatment of SOD1-ALS since 2023. Previously, SOD1 patients in Germany had the opportunity to participate in an open access programme run by Biogen. Around 40 patients across Germany have already been successfully treated in this programme. In May 2024, the EMA has now also granted marketing authorisation for Europe. This means that the open access programme will end in June 2024 and the drug will be available in the classic form from 1 July 2024.

Similar to ASO therapy for spinal muscular atrophy (Spinraza®), the drug must be injected into the spinal canal (intrathecal administration). This is done 3 times in the first month, then at 28-day intervals. Experience to date in the German network shows that the treatment is well tolerated by most patients.

Article on the pathophysiology of ALS

New article published on the impact of defective DNA repair on the formation of FUS protein aggregates in the cytoplasm of motor neurons.

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RELYVRIO at ALS

On 29 September 2022, the US Food and Drug Administration (FDA) approved AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) for the treatment of adults with amyotrophic lateral sclerosis (ALS) under the brand name RELYVRIO.

Marketing authorisation was applied for in the European Union (EU) and initially not granted, with the reasoning that the results of the Phase III trial (PHOENIX trial) should be awaited first. These were not available until early 2024. The Phase III trial was negative in all study objectives.

As a result, it was clear that RELYVRIO would not be authorised in the European Union (EU). The company Amylyx also withdrew the marketing authorisation in the countries where it had already been granted (USA, Canada), meaning that this therapy is no longer available and cannot be recommended based on the current study situation.