Search Results for "diagnosis"

This infographic discusses the clinical and demographic presentation of an AHP patient and how to assess for AHP diagnosis.

This educational module will illustrate patients with AHP, describe common AHP symptoms and summarize how patients present to the various specialties involved in managing these patients

This infographic discusses the clinical and demographic presentation of a PH1 patient and how to assess for PH1 diagnosis

The handout details potential clinical characteristics that pediatric patients may present with which should warrant suspicion for PH1.

This slide deck provides disease education on ATTR amyloidosis, focusing on the musculoskeletal manifestations associated with the disease and their importance for early diagnosis and patient care.

This slide deck presents of overview of ATTR amyloidosis, covering pathophysiology, disease manifestations and the clinical burden of ATTR-CM.

This handout provides an overview of ATTR manifestations and the diagnostic tools that can be used to assess their presentation.

V122I is the most common variant of hATTR amyloidosis in the United States and is most prevalent in patients of African American descent. Compared to other variants, patients with the V122I variant experience significant disease burden and poorer outcomes.

AHP Diagnosis

ASPIRE: Amyloidosis Industry Collaborative convenes leading companies to address some of the most urgent issues faced by the amyloidosis community. Through partnership we are driven to shift the discourse and demonstrate what can be done when leaders work together on behalf of rare disease patients. ASPIRE aims to accelerate progress in the areas of diagnosis, care, and addressing health disparities through collaborative project design and shared resources, arriving at solutions that address common challenges and unmet need.

Virtual interactive clinic offers quarterly 1-hour sessions focused on AL (light chain) and ATTR (transthyretin) amyloidosis, two underrecognized but increasingly manageable causes of heart failure and multisystem disease.

Standard response letter on the use of givosiran in patients with a prior history of hemin prophylaxis from the ENVISION study.

ATTR-CM HELIOS-B Data Summary deck provides a comprehensive overview of the HELIOS-B results. Also includes information on ATTR-CM and the MOA of RNAi therapeutics.

Infographic companion to the article by Sato et al, "Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis", published in the journal Amyloid, 2023.

Standard response letter on givosiran and pediatric use. This medical information response contains clinical data from the ENVISION study, ELEVATE registry, and published case reports.

Standard response letter on the use of givosiran in patients with pre-existing renal impairment.