Active clinical trials for "Amyloidosis, Familial"

The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.

The purpose of this study is to determine if diflunisal can prevent progressive lower leg nerve damage in patients with familial amyloidosis polyneuropathy. Funding Source - FDA Office of Orphan Products Development (OOPD); National Institute of Neurological Disorders and Stroke (NINDS)

Efficacy of 308-nm excimer laser for primary localized cutaneous amyloidosis treatment in Asians, pilot study.

This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers. The results will be used to describe the emotional, physical, and financial impact of having TTR-FAP or caring for someone who has the disease.

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).

Amyloidosis is a serious systemic disease. Cardiac involvement has a great impact on prognosis and can occur in its three main forms: acquired monoclonal light chain, hereditary transthyretinal and senile form. The physiopathogenesis basically results from the deposition of an abnormal protein (amyloid) with toxic properties to the myocyte. The scope of this study will be a hereditary transthyretinal amyloidosis (hATTR). It is known that amyloidotic cardiomyopathy due to transthyretin deposit is an underdiagnosed cause of heart failure in adults, being an important differential diagnosis of diseases that manifest with increased myocardial thickness, such as hypertrophic cardiomyopathy or myocardial hypertrophy that accompanies the different degrees of aortic valve stenosis. The human gut microbiota is immensely diverse. It is estimated at around 100 trillion microorganisms, including bacteria, fungi and viruses. The microbiota of each individual is unique and determined by genetic factors such as age, type of delivery, use of antibiotics and diet. Recent data point to the hypothesis that the resilience of the intestinal microbiota plays a role in the process of disease development and health restoration.