Genomic Translation for ALS Care
Condition: Amyotrophic lateral sclerosis (ALS)
Key Inclusion Criteria
- At least 18 years old
- Diagnosis of familial or sporadic ALS, or those with primary lateral sclerosis or progressive bulbar/muscular atrophy forms of motor neuron disease
- Willing to return to clinic site (or another participating center) for follow-up care
- Capable of providing informed consent and following study procedures
Key Exclusion Criteria
- Invasive ventilation (i.e., tracheostomy) in place
- Non-invasive ventilation dependent (defined as >22 hours per day)
- Known HIV, chronic hepatitis B or hepatitis C
Full Study Name
Genomic Translation for ALS Care (GTAC) (IRB no. 45860)
This study focuses on individuals who have been diagnosed with amyotrophic lateral sclerosis (ALS) or a related motor neuron disease (MND) such as primary lateral sclerosis, progressive muscular atrophy, or progressive bulbar palsy. The purpose of the study is to collect specimens and data for future analyses. Participants' specimens and data will be used to look for genetic changes and gene expression profiles that help determine why a person develops ALS and related MND (referred also to ALS/MND) and why their symptoms present and progress with a particular pattern. Specimens will also be used for future studies to create cells called induced pluripotent stem cells (iPSC).
ALS/MND is caused by the progressive decline of motor neurons. However, every individual is affected differently. Uncovering factors causing ALS variability is likely to provide clues about how to better diagnose and treat the disease.