[HTML][HTML] Treatment of Fabry's disease with the pharmacologic chaperone migalastat

DP Germain, DA Hughes, K Nicholls… - New england journal …, 2016 - Mass Medical Soc
Background Fabry's disease, an X-linked disorder of lysosomal α-galactosidase deficiency,
leads to substrate accumulation in multiple organs. Migalastat, an oral pharmacologic …

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT …

DA Hughes, K Nicholls, SP Shankar… - Journal of medical …, 2017 - jmg.bmj.com
Background Fabry disease is an X-linked lysosomal storage disorder caused by GLA
mutations, resulting in α-galactosidase (α-Gal) deficiency and accumulation of lysosomal …

[HTML][HTML] Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable …

DP Germain, K Nicholls, R Giugliani, DG Bichet… - Genetics in …, 2019 - Elsevier
Purpose Outcomes in patients with Fabry disease receiving migalastat during the phase 3
FACETS trial (NCT00925301) were evaluated by phenotype. Methods Data were evaluated …

The adult rat olfactory system expresses microtubule-associated proteins found in the developing brain

C Viereck, RP Tucker, A Matus - Journal of Neuroscience, 1989 - Soc Neuroscience
We have compared the expression and localization of neuronal microtubule-associated
proteins (MAPs) MAP5, MAP2, and tau in the adult rat olfactory system and cerebral cortex …

An analysis of the impact of FDA's guidelines for addressing cardiovascular risk of drugs for type 2 diabetes on clinical development

C Viereck, P Boudes - Contemporary clinical trials, 2011 - Elsevier
We examined the impact of FDA's 2008 guidelines for addressing cardiovascular risks of
new therapies for type 2 diabetes on clinical trials. We focused on the new class of incretin …

The expression of phosphorylated and non-phosphorylated forms of MAP5 in the amphibian CNS

C Viereck, A Matus - Brain research, 1990 - Elsevier
MAP5 is a microtubule-associated protein that in rat and quail is more abundant in the
developing than in the adult brain. Previous studies in our laboratory have shown that MAP5 …

[PDF][PDF] An investigation into multifaceted mechanisms of action of allantoin in wound healing

A Paller, R Nardi, H Do, A Reha, C Viereck… - J Am Acad …, 2017 - amicusrx.com
• While different allantoin-containing preparations have been used clinically to study its
therapeutic effects in wound healing, its mechanisms of action are not known with certainty• …

A “bridging”(safety/tolerance) study of besipirdine hydrochloride in patients with Alzheimer's disease

JJ Sramek, C Viereck, FJ Huff, T Wardle, J Hourani… - Life sciences, 1995 - Elsevier
Besipirdine hydrochloride is a novel compound with cholinergic and adrenergic activity
being investigated as a treatment for Alzheimer's disease (AD). The pharmacodynamics of …

An analysis of current pharmaceutical industry practices for making clinical trial results publicly accessible

C Viereck, P Boudes - Contemporary clinical trials, 2009 - Elsevier
We compared the clinical trial transparency practices of US/European pharma by analyzing
the publicly-accessible clinical trial results databases of major drugs (doripenem …

Microtubule-associated proteins in cerebellar morphogenesis

A Matus, RP Tucker, C Viereck - The Cerebellum Revisited, 1992 - Springer
In his studies of the cerebellum, Ramón y Cajal (1888, 1960) noted, with characteristic
clarity, the major features of morphogenesis in the various cell types. Many of his …