Epigenetics of neurodegenerative diseases

FDA Approves Oral Treatment for Spinal Muscular Atrophy

Retrieved on: 
Friday, August 7, 2020

This is the second drug and the first oral drug approved to treat this disease.

Key Points: 
  • This is the second drug and the first oral drug approved to treat this disease.
  • SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control movement.
  • The efficacy of Evrysdi for the treatment of patients with infantile-onset and later-onset SMA was evaluated in two clinical studies.
  • After 23 or more months of treatment, 81% of patients were alive without permanent ventilation, which is a noticeable improvement from typical disease progression without treatment.

Scholar Rock Presents Clinical and Preclinical Data for SRK-015 at the 2020 Virtual Cure SMA Research and Clinical Care Meeting

Retrieved on: 
Friday, June 12, 2020

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced two presentations as part of the 2020 Virtual Cure SMA Research and Clinical Care Meeting.

Key Points: 
  • Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced two presentations as part of the 2020 Virtual Cure SMA Research and Clinical Care Meeting.
  • SRK-015 is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Preclinical SMA mouse model studies have demonstrated that selective inhibition of myostatin may have therapeutic potential in improving muscle strength(2).
  • A Phase 2 clinical trial in patients with Type 2 and Type 3 SMA is ongoing (NCT03921528).

Emerging Therapeutics for Neurodegenerative Diseases Part II: Huntington's Disease and Amyotrophic Lateral Sclerosis (HD & ALS)

Retrieved on: 
Monday, October 7, 2019

The most common denominator among neurodegenerative diseases, in addition to nerve cell loss is inflammation.Although, a series of mutant genes and environmental toxins are related to neurodegenerative disorders, the causal mechanisms remain poorly understood.

Key Points: 
  • The most common denominator among neurodegenerative diseases, in addition to nerve cell loss is inflammation.Although, a series of mutant genes and environmental toxins are related to neurodegenerative disorders, the causal mechanisms remain poorly understood.
  • Overall, neurodegenerative diseases affect approximately 30 million individuals worldwide, according to the World Health Organization (WHO).
  • Factors Contributing to Neurodegenerative DiseasesThe factors contributing to the onset of neurodegenerative diseases are multiple.
  • Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and amyotrophic lateral sclerosis (ALS) diseases are well-established as the consequence of misfolding and dysfunctional trafficking of proteins.