Andrew Heim
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View article: Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis
Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis Open
Instrumental to the progress of the trial has been the collaboration between site teams, patient partnership and adaptability.
View article: A 40‐week phase <scp>2B</scp> randomized, multicenter, double‐blind, placebo‐controlled study evaluating the safety and efficacy of memantine in amyotrophic lateral sclerosis
A 40‐week phase <span>2B</span> randomized, multicenter, double‐blind, placebo‐controlled study evaluating the safety and efficacy of memantine in amyotrophic lateral sclerosis Open
Introduction Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease with no known cure, limited treatment options with minimal benefits, and significant unmet need for disease modifying therapies. Aims This …
View article: Safety and tolerability of phenylbutyrate in inclusion body myositis
Safety and tolerability of phenylbutyrate in inclusion body myositis Open
Introduction
Phenylbutyrate (PBA) showed positive effect on the muscle cell model of Inclusion Body Myositis (IBM) by improving lysosomal activity, ameliorating consequences of impaired autophagy, and decreasing vacuolization. This provid…
View article: Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial
Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial Open
US Food and Drug Administration Office of Orphan Products Development and Orphazyme.
View article: Identification of 5-HT<sub>2A</sub>Receptor Signaling Pathways Responsible for Psychedelic Potential
Identification of 5-HT<sub>2A</sub>Receptor Signaling Pathways Responsible for Psychedelic Potential Open
Summary Serotonergic psychedelics possess considerable therapeutic potential. Although 5-HT 2A receptor activation mediates psychedelic effects, prototypical psychedelics activate both 5-HT 2A -Gq/11 and β-arrestin2 signaling, making their…
View article: Timing of Decremental Response During Repetitive Nerve Stimulation in Myasthenia Gravis
Timing of Decremental Response During Repetitive Nerve Stimulation in Myasthenia Gravis Open
Background: A decrement >10% detected during repetitive nerve stimulation (RNS) is supportive of considering a diagnosis of myasthenia gravis (MG). Several studies have found that most of this decrement is seen between 4 to 6 min post-exer…
View article: Comparative Effectiveness of Intravenous Immunoglobulin (IVIg) and Subcutaneous Immunoglobulin (SCIg) versus Historical controls in Management of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Comparative Effectiveness of Intravenous Immunoglobulin (IVIg) and Subcutaneous Immunoglobulin (SCIg) versus Historical controls in Management of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Open
The reviewers appreciated the question we were trying to address although they were not very impressed that we were willing to tackle the comparison of IVIg to SCIg.We clearly did not explain the issue well as one reviewer said we did not …
View article: The Early History of Arimoclomol for Inclusion Body Myositis
The Early History of Arimoclomol for Inclusion Body Myositis Open
View article: A Patient Activities of Daily Living Scale for Amyotrophic Lateral Sclerosis
A Patient Activities of Daily Living Scale for Amyotrophic Lateral Sclerosis Open
Background: Motor neuron disorders are rare, progressive neurodegenerative diseases which affect multiple domains of motor function. The ability to assess function from home using an electronic medical record (EMR) would facilitate pragmat…
View article: Amyloid Myopathy as an Inclusion Body Myositis Mimic
Amyloid Myopathy as an Inclusion Body Myositis Mimic Open
Introduction: Amyloid myopathy is a rare presentation of systemic amyloidosis. Amyloid myopathy can be initially misdiagnosed as sporadic inclusion body myositis (IBM).
Methods: We report 4 cases of amyloid myopathy clinically mimicking i…
View article: Clinical Findings in Isolated Bulbar Amyotrophic Lateral Sclerosis
Clinical Findings in Isolated Bulbar Amyotrophic Lateral Sclerosis Open
Background: Isolated bulbar amyotrophic lateral sclerosis (IBALS) is a regional variant of amyotrophic lateral sclerosis (ALS) with weakness restricted to the bulbar muscles for at least 2 years, and slower progression than generalized ALS…
View article: CIDP Diagnostic Criteria and Response to Treatment
CIDP Diagnostic Criteria and Response to Treatment Open
Introduction: Diagnostic criteria for CIDP have been proven useful for clinical trials. However, use of these criteria in clinics has been limited by time constraints and unknown usefulness in predicting outcomes. Methods: A retrospective …
View article: Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS
Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS Open
Serum NfL may be considered a clinically validated prognostic biomarker for ALS. Serum NfL (and perhaps pNfH), quantified using the Simoa assay, has potential utility as a pharmacodynamic biomarker of treatment effect.
View article: Methotrexate Polyglutamation in a Myasthenia Gravis Clinical Trial
Methotrexate Polyglutamation in a Myasthenia Gravis Clinical Trial Open
Introduction. Methotrexate (MTX) is an immunosuppressive and anti-inflammatory drug used to treat rheumatoid arthritis (RA) and other autoimmune conditions. MTX is transported into cells, where glutamate moieties are added and is ret…
View article: Self-Prompted Discrimination and Operant Control of EEG Alpha
Self-Prompted Discrimination and Operant Control of EEG Alpha Open
EEG state discrimination studies may contribute to understanding the role of awareness in physiological self-regulation, but many individuals learn the existing paradigm very slowly. In this study, a self-prompted discrimination paradigm, …