Since 2007 Dr. Mandrioli is the chief of the Multidisciplinary Centre for MND in Modena, Italy, a referral centre for diagnosis and treatment of MND and follows 250 patients every year. She created and has been coordinating the population based Registry of ALS in Modena since 2000, and in Emilia Romagna Region (4.4 million inhab.) since 2009. This Registry collects data on patients from 17 Italian neurological centres. She carried out several epidemiological studies on ALS in Italy. She took part, together with ITALSGEN Consortium, in several studies on genetics of ALS that led to the isolation of new genes (including VCP, C9ORF72, MATRIN3, KIF5A), and to pharmacological clinical trials in ALS. She coordinates clinical trials on ALS (currently: RAP-ALS, co-ALS, FETR-ALS). She was coordinator of the Italian Study Group on MND from 2013 to 2017. She has published in over 100 peer-reviewed scientific journals and her work is highly cited by other scientists.
Amylyx Pharmaceuticals Inc is conducting a phase 3 trial to determine the safety and efficacy of their compound ‘AMX0035’ for the treatment of ALS.
Ferrer is conducting a phase 3 trial to evaluate the safety and potential efficacy of an investigational drug for people with Amyotrophic Lateral Sclerosis (ALS).
In the COURAGE-ALS study, we are investigating the efficacy and safety of the drug ‘reldesemtiv’ for the treatment of ALS. This clinical trial is sponsored by Cytokinetics.
In this phase 2 study, we are investigating the safety and efficacy of the drug pegcetacoplan for the treatment of ALS. This trial is sponsored by Apellis Pharmaceuticals.
RT001 in Amyotrophic Lateral Sclerosis
In this phase 2 trial, we will study the safety and efficacy of the compound RT001 in people living with ALS. This clinical trial is sponsored by Retrotope Inc.
This trial will evaluate the efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment for people with ALS.