Ultragenyx Pharmaceuticals has shared data from two cohorts taking part in an on-going study involving an investigational gene therapy for ornithine transcarbamylase deficiency. For more information, you can view the…
Avrobio has received no objection from Health Canada for their clinical trial application for their Phase 1/2 study of an investigational gene therapy for Gaucher disease. Following this news, Avrobio…
A study of an investigational drug called ZX008 in patients who have refractory Lennox-Gastaut syndrome has produced promising results, according to Zogenix. For more detailed information, you can view…
Researchers are looking for participants to take part in a study investigating whether a mobile app could benefit patients with multiple sclerosis. For more information about this study and how…
Initial results from a clinical trial investigating the effects of an experimental drug called BCX7353 in patients with hereditary angioedema suffering from acute attacks. For more detailed information, you can…
The drug minocycline is a tetracycline antibiotic. Previous research using mouse models has indicated that it may have anti-inflammatory and antiapoptotic (prevents cell death) properties and may benefit some neurological…
The speciality pharmaceutical company Tikomed has announced that the UK Medicines and Healthcare Products Regulatory Agency has granted the company permission to go ahead with a Phase 2 clinical study.…
A recent study into Periodic Fever, Aphtous stomatitis, Pharyngitis and Adenitis (PFAPA) syndrome investigated the impact of the condition on children and found that is was associated with a reduction…
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