Publications by authors named "S A Eraly"
Background: Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a fatal disease, caused by misfolded transthyretin depositing as amyloid fibrils in the heart. Because disease progression is common, practical and sensitive methods are needed to monitor patients and optimize treatment decisions. Outpatient worsening heart failure (HF) (oral loop diuretic intensification or initiation) is simple to assess and has been shown to be prognostic of mortality in patients with ATTR-CM.
View Article and Find Full Text PDFArticle Synopsis
- Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a serious, progressive disease, and vutrisiran is a new treatment that works by reducing the production of transthyretin in the liver.
- In a double-blind trial involving 655 patients, those receiving vutrisiran had a lower risk of death and cardiovascular events compared to those on placebo, demonstrating significant efficacy.
- Vutrisiran also improved patient outcomes, showing less decline in walking distance and quality of life measurements over the study period compared to placebo.
Article Synopsis
- The study investigates the safety, tolerability, and pharmacokinetics of BIIB078, an investigational treatment targeting the genetic cause of amyotrophic lateral sclerosis (ALS) linked to the C9orf72 gene mutation.
- The trial involved 106 participants with C9orf72-associated ALS, who were randomly assigned to receive varying doses of BIIB078 or a placebo over a treatment period of three to six months.
- Results showed that all participants experienced at least one adverse event, mostly mild or moderate, indicating that while BIIB078 did pose some risks, it did not lead to a high rate of treatment discontinuation.
Phosphorylated neurofilament heavy subunit (pNfH) has been recently identified as a promising biomarker of disease onset and treatment efficacy in spinal muscular atrophy (SMA). This study introduces a quantitative systems pharmacology model representing the SMA pediatric scenario in the age range of 0-20 years with and without treatment with the antisense oligonucleotide nusinersen. Physiological changes typical of the pediatric age and the contribution of SMA and its treatment to the peripheral pNfH levels were included in the model by extending the equations of a previously developed mathematical model describing the neurofilament trafficking in healthy adults.
View Article and Find Full Text PDFIn recent years the neurobiological underpinnings of catatonia have been an emerging area of interest. Catatonia is frequently encountered in mood disorders, neurological disorders and systemic illnesses. Furthermore, the manifestation of catatonia in autoimmune disorders such as NMDA receptor antibody encephalitis and thyroiditis reinforces its neuropsychiatric nature.
View Article and Find Full Text PDF