In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.
This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using 2 different groups of study research centers that help provide clinical care for participant with ALS. These groups are in Europe and the United States and are called:
* the Precision-ALS programme * the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC)
The main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups.
The main question researchers want to answer in this study is:
* What are the characteristics of the participants in this study? * How many participants had serious adverse events (SAEs), including ones that affect the brain, spinal cord, or nerves?
An adverse event is a health problem that may or may not be caused by a drug during the study. An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
Researchers will also learn more about:
* How many participants develop other health conditions or become pregnant, including how the pregnancy turned out * Why and when participants stopped treatment
This study will be done as follows:
* Participants will be screened to check if they can join the study. * Data from the participants' regular visits to their clinic will be collected based on which study research center they are in. * Each participant will be in the study until they decide to leave or until death. Currently, the study is planned to last at least 7 years.
Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.
Data collected while a person with SOD1-ALS is participating in an interventional clinical trial (with tofersen or any other investigational medicinal product) will be excluded.
Participants with SOD1-ALS enrolled in TRICALS network's Precision-ALS programme with data from participating clinical centers across multiple European countries or at ALS/MND NHC with data from participating clinical centers in the United States (tofersen users and tofersen non-users).
Cohort
Prospective
Tofersen
DrugAdministered as specified in the treatment arm.
Baseline Demographic: Age
Baseline Demographic: Participant Sex
Baseline Demographic: Race/Ethnicity
Baseline Demographic: Weight
Number of Participants With New Comorbid Conditions
Number of Participants With Pregnancy and Pregnancy Outcomes
Number of Participants With Reported Treatment Discontinuation
Number of Participants With Reported Reasons for Treatment Discontinuation
