Mark B. Bromberg
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View article: D.4 International consensus recommendations for the management of glucocorticoid complications in neuromuscular disease
D.4 International consensus recommendations for the management of glucocorticoid complications in neuromuscular disease Open
Background: Adverse effects and risks associated with glucocorticoid (GC) treatment are frequently encountered in immune-mediated neuromuscular disorders. However, significant variability exists in the management of these complications. Ou…
View article: ALSUntangled #75: Portable neuromodulation stimulator therapy
ALSUntangled #75: Portable neuromodulation stimulator therapy Open
Spurred by patient interest, ALSUntangled herein examines the potential of the Portable Neuromodulation Stimulator (PoNS™) in treating amyotrophic lateral sclerosis (ALS). The PoNS™ device, FDA-approved for the treatment of gait deficits i…
View article: A prospective observational study of decision‐making by patients with amyotrophic lateral sclerosis upon recommendation for PEG enteral feeding tubes
A prospective observational study of decision‐making by patients with amyotrophic lateral sclerosis upon recommendation for PEG enteral feeding tubes Open
Objective To understand challenges surrounding acceptance of a percutaneous endoscopic gastroscopic enteral feeding tube by patients with amyotrophic lateral sclerosis: a prospective observational study. Methods This was a prospective obse…
View article: ALSUntangled #78: Zinc
ALSUntangled #78: Zinc Open
ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). In this review, we assess the utilization of dietary zinc supplements for modulating ALS pathology and progression. Stud…
View article: Evaluating Range of Motion of Two Prominent Neck Support Devices for Daily Activities
Evaluating Range of Motion of Two Prominent Neck Support Devices for Daily Activities Open
Neck muscle weakness causes the inability to raise and move the head, leading to fatigue, neck pain, and a "head-on-chest" posture (dropped head syndrome) in severe cases, which significantly affects quality of life. Static neck collars ar…
View article: Preliminary Study on Effects of Neck Exoskeleton Structural Design in Patients With Amyotrophic Lateral Sclerosis
Preliminary Study on Effects of Neck Exoskeleton Structural Design in Patients With Amyotrophic Lateral Sclerosis Open
Neck muscle weakness due to amyotrophic lateral sclerosis (ALS) can result in dropped head syndrome, adversely impacting the quality of life of those affected. Static neck collars are currently prescribed to hold the head in a fixed uprigh…
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: ALSUntangled #71: Nuedexta
ALSUntangled #71: Nuedexta Open
Nuedexta is a combination of dextromethorphan hydrobromide and quinidine sulfate and was approved by the Food and Drug Administration (FDA) in 2010 to treat pseudobulbar affect (PBA). There have since been anecdotal case reports of bulbar …
View article: ALSUntangled #70: caffeine
ALSUntangled #70: caffeine Open
ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here, we review caffeine which has plausible mechanisms for slowing ALS progression. However, pre-clinical studies are c…
View article: ALSUntangled #68: ozone therapy
ALSUntangled #68: ozone therapy Open
ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here we review ozone therapy. Ozone therapy has possible mechanisms for slowing ALS progression based on its antioxidant…
View article: ALSUntangled #66: antimycobacterial antibiotics.
ALSUntangled #66: antimycobacterial antibiotics. Open
Several infections have been associated with motor neuron diseases resembling ALS, including species of viruses, bacteria, and parasites. Mycobacterium avium subspecies paratuberculosis (MAP), most known for its probable etiologic associat…
View article: Expanding Clinical Spectrum of <i>C9ORF72</i> -Related Disorders and Promising Therapeutic Strategies
Expanding Clinical Spectrum of <i>C9ORF72</i> -Related Disorders and Promising Therapeutic Strategies Open
In 2011, a pathogenic hexanucleotide repeat expansion in the C9ORF72 gene was discovered to be the leading genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Before this, the C9ORF72 gene and its protei…
View article: ALSUntangled #64: butyrates
ALSUntangled #64: butyrates Open
ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). Here we review butyrate and its different chemical forms (butyrates). Butyrates have plausible mechanisms for slowing AL…
View article: <i>MFN2</i>Influences Amyotrophic Lateral Sclerosis Pathology
<i>MFN2</i>Influences Amyotrophic Lateral Sclerosis Pathology Open
Objective To better understand the pathology of amyotrophic lateral sclerosis, we used sequence data from patients seen at the University of Utah to identify novel disease-associated loci. We utilized both in vitro and in vivo studies to d…
View article: ALSUntangled #63: ketogenic diets
ALSUntangled #63: ketogenic diets Open
ALSUntangled reviews alternative and off label treatments with a goal of helping patients make more informed decisions about them. Here we review ketogenic diets. We shows that these have plausible mechanisms, including augmenting cellular…
View article: Pathogenic Effect of <i>TP73</i> Gene Variants in People With Amyotrophic Lateral Sclerosis
Pathogenic Effect of <i>TP73</i> Gene Variants in People With Amyotrophic Lateral Sclerosis Open
Together, these results strongly suggest that variants in TP73 correlate with risk for ALS and indicate a role for apoptosis in ALS disease pathology.
View article: Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis
Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis Open
ClinicalTrials.gov Identifier: NCT03315130.
View article: Loss of<i>TP73</i>function contributes to amyotrophic lateral sclerosis pathogenesis
Loss of<i>TP73</i>function contributes to amyotrophic lateral sclerosis pathogenesis Open
Much remains unknown about the genetics and pathophysiology underlying the neurodegenerative disease amyotrophic lateral sclerosis (ALS). We analyzed exome sequences from a cohort of 87 sporadic ALS (SALS) patients and 324 healthy individu…
View article: Voltage-gated calcium channel autoimmune cerebellar degeneration
Voltage-gated calcium channel autoimmune cerebellar degeneration Open
Autoimmune cerebellar degeneration associated with VGCC autoantibodies may precede onset of LEMS and may improve with immunosuppressive treatment. Binding of anti-VGCC antibodies to Purkinje cells in cerebellar slice cultures may be follow…
View article: What's in the Literature?
What's in the Literature? Open
In this issue, we review the question of geographic location and head trauma as a contributing factor for amyotrophic lateral sclerosis. We also discuss the utility of ganglioside antibody testing and present new insights on respiratory fu…
View article: Relationship of creatine kinase to body composition, disease state, and longevity in ALS
Relationship of creatine kinase to body composition, disease state, and longevity in ALS Open
Our objective was to explore if creatine kinase (CK) levels correlate with survival in amyotrophic lateral sclerosis (ALS), and whether a correlation is independent of other well-studied predictors such as location of onset, gender, age, f…