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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.

Standard response letter on vutrisiran and a post-hoc analysis of the HELIOS-B monotherapy population censoring data following tafamidis initiation.

Standard response letter on vutrisiran and pregnancy & lactation. This letter contains information on preclinical toxicology studies and the HELIOS-A and HELIOS-B studies.

Standard response letter on vutrisiran and data from patients who underwent serial CMR and participated in HELIOS-B.

Standard response letter on the phase 1 study of mivelsiran.

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 vutrisiran in patients who had progressed on tafamidis at baseline in HELIOS-B.

Standard response letter on the use of vutrisiran in patients who have had a major surgery or anticipated surgery.

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

Standard response letter containing pre-clinical data on the tissue distribution of vutrisiran.

Standard response letter containing pre-clinical data on the tissue distribution of patisiran.

This infographic discusses the study design, objectives, key patient criteria, and planned countries for the BONAPH1DE observational study.

Standard response letter on vutrisiran and gastrointestinal events in the HELIOS-B study.

Standard response letter on vutrisiran and patients with NYHA Class III HF and NAC Stage 3 or NHYA Class IV HF.

Standard response letter on the use of vutrisiran and vaccines.

Standard response letter on mortality and survival data from the HELIOS-B study of vutrisiran.

Standard response letter on transitioning between patisiran and vutrisiran therapy.

Standard response letter on vutrisiran and use in patients with renal impairment.

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

Standard response letter on givosiran and use in patients with pre-existing kidney impairment from the ENVISION study.

Standard response letter on lumasiran and dialysis. This medical information response contains clinical data from the ILLUMINATE-C study and published case reports.

Standard response letter on the mechanism of action of lumasiran.

The porphyria burden assessment (PBA) is a digitally accessible patient questionnaire intended to capture patients’ lived experiences of acute hepatic porphyria (AHP), focused on the chronic symptom burden associated with the condition and the impact of AHP on daily life

Standard response letter on vutrisiran and thyroid function.

Standard response letter on use of vutrisiran across baseline heart failure disease severity of patients enrolled in HELIOS-B.