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LGMD1B
Clinical trial pipeline · Data from ClinicalTrials.gov
See which LGMD1B trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which LGMD1B trials you may qualify forThis is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset P…
Laminopathies and emerinopathies are complex group of rare disorders due to mutations in A-type lamins (LMNA) and Emerin (EMD) genes. Among them, disorders affe…
The goal of this clinical trial is to know the effect of multicomponent exercise in frail elderly in community to aging hallmarks. The main question\[s\] it aim…
The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electron…
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD). In this expanded acces…
The purpose of this study is to assess the pharmacokinetics, excretion, mass balance and metabolism of PF-07265803 (formerly known as ARRY-371797) in approximat…
This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C…