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Neuromuscular; Disorder, Hereditary
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Neuromuscular; Disorder, Hereditary trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Neuromuscular; Disorder, Hereditary trials may be worth asking aboutNorth America
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The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life,…
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1)…
Background: Cholangiocarcinoma (CCA) is an aggressive cancer of the bile ducts. People with CCA have few treatment options and poor survival. Researchers want…
Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-…
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients treated with Koselugo (…
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participant…
The aim of this study is to investigate the relationship between Pes Planus and Piriformis Syndrome. The study will be conducted on individuals aged 18-65 years…
The ability of an individual to conceive some alternative representations and to behave in a flexible manner would emerge from preschool age and drastically imp…