Advanced Practice Providers: ALS Therapeutics: New Treatments & New Approaches-ALS Therapeutics: New Treatments & New Approaches

Video Transcription

Video Summary

A recent ALS session provided an extensive overview of the disease, covering pathophysiology, current therapeutic landscapes, and clinical trials. The session also included significant insights from a range of experts into diagnosis and treatment challenges. A tribute was paid to Dr. Lisa Krivikas, a pioneering figure in ALS care, whose personal experience as a caregiver and patient highlighted the emotional and professional challenges of ALS.<br /><br />Over the years, innovative therapies like antisense oligonucleotides (ASO) targeting the SOD1 mutation have shown promise, albeit mixed results in clinical trials. The discussion extended to include broader genetic and sporadic forms of ALS, including the complex pathophysiology of the C9orf72 gene. Despite progress in genetic understanding, ALS diagnosis remains challenging. Patients often experience delays because of missed ALS signs and misdiagnoses, averaging 12 to 15 months from symptom onset to diagnosis. Efforts to improve early diagnosis include clinician education tools and national recommendations for integrated ALS care systems modeled after cancer care centers.<br /><br />The session also emphasized the importance of multidisciplinary care in improving patient outcomes. Rehabilitation through exercise, respiratory support, and pain management for shoulder issues was recommended to enhance quality of life. ALS rehabilitation involves a team approach, notably integrating neurologists and physiatrists to customize care plans. The potential benefits and safety of exercise in ALS were highlighted, with rehabilitation shown to be a powerful intervention in prolonging and improving patients' quality of life.<br /><br />Overall, ALS care continues to advance with more target-driven therapies and strategies to offer better outcomes for patients.

Asset Subtitle

Timestamps of interest:
- Discussion on ALS therapy research: ~3:30 minute mark
- Tool to identifying patients earlier ~15 minute mark
- EMG ~ 37 minute mark
- Exercise in ALS ~ 1 hour 7 min mark
- Shoulder pain in ALS ~ 1 hour 17 minute mark
- Discussion of Advanced ALS and MDC - ~1 hour 21 minute mark

Asset Caption

There are 2 questions in the Post Exam. Please use these questions to help test your knowledge after viewing the course. There is not a minimum score needed to pass.

Keywords

ALS

genetic basis

therapeutic pipeline

late-stage testing

AMX0035

Healy ALS Platform Trial

collaboration

amyotrophic lateral sclerosis

ALS research

pathophysiology

therapeutic strategies

clinical trials

antisense oligonucleotide therapies

SOD1 gene mutations

Valor study

diagnostic tools

genetic panel testing

multidisciplinary approach

rehabilitation medicine

exercise and rehabilitation

integrated care systems

therapeutic landscapes

antisense oligonucleotides

SOD1 mutation

C9orf72 gene

diagnosis challenges

multidisciplinary care

rehabilitation

exercise

Dr. Lisa Krivikas