Filtered By:
Condition: Spinal Muscular Atrophy

This page shows you your search results in order of relevance.

Order by Relevance | Date

Total 31 results found since Jan 2013.

Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) information page compiled by the National Institute of Neurological Disorders and Stroke (NINDS).
Source: NINDS Disorders: National Institute of Neurological Disorders and Stroke - October 26, 2014 Category: Neurology Source Type: research

Non-invasive assessment of motor unit activation in relation to motor neuron level and lesion location in stroke and spinal muscular atrophy
The results suggest, that for stroke, information from the brain is modified thereby resulting in motor units firing at their natural frequency. Thus, high spatial resolution electromyography and the chosen parameters facilitate non-invasive, objective differentiation and analysis of the activation patterns of motor units in neuromuscular disorders.
Source: Clinical Biomechanics - May 26, 2020 Category: Orthopaedics Authors: Sybele E. Williams, Kathrin C. Koch, Catherine Disselhorst-Klug Source Type: research

Safety and Tolerability of Systemic Beta-2-Adrenergic AgonistAlbuterol as Pharmacological Therapy in Non-Invasive Ventilation NIV -supported Amyotrophic Lateral SclerosisALSPatients with Chronic Respiratory Failure (P4.079)
CONCLUSIONS:Systemic albuterol is 94.2% tolerable when used in NIV-supported ALS patients.M NIV-supported ALS patients more commonly required albuterol.VC was maintained at 3 months without change but not at 6 months in albuterol-treated NIV-supported ALS patients.Further studies are required on pharmacologically enhancing the treatment of NIV-supported ALS patients.Study Supported by:Carolinas ALS Research Fund/Pinstripes Fund/Carolinas Garden of Hope/Carolinas Healthcare FoundationDisclosure: Dr. Brooks has received personal compensation for activities with Biogen Idec, Avanir Pharmaceuticals, Acorda Therapeutics, Cytoki...
Source: Neurology - April 9, 2014 Category: Neurology Authors: Brooks, B., Kandinov, B., Langford, V., Lindblom, S., Sanjak, M., Wright, K., Ward, A., Holsten, S., Fischer, M., Lucas, N., Smith, N., Nichols, M., Lary, C., Nemeth, J., Russo, P., Bockenek, W., Bravver, E., Desai, U., Story, J. S., Pacicco, T. Tags: ALS: Trials and Biomarkers Source Type: research

Serum Creatinine, a Biomarker for Muscle Mass in Amyotrophic Lateral Sclerosis (ALS), Predicts Loss of Ambulation Measured by ALS Functional Rating Scale-Revised Walking Item Score (ALSFRS-Rw) (P4.085)
CONCLUSIONS:Decrease in serum creatinine significantly predicted change in ambulation measured by ALSFRS-Rw longitudinally.Further analysis is required to determine whether leg function alone and leg muscle mass are the major determinants of serum creatinine in ALS patients over the course of the disease.Study Supported by:Carolinas ALS Research Fund/Pinstripes Fund/Carolinas Garden of Hope/Carolinas Healthcare FoundationDisclosure: Dr. Brooks has received personal compensation for activities with Biogen Idec, Avanir Pharmaceuticals, Acorda Therapeutics, Cytokinetics, Synapse, and the National Institute of Neurological Dis...
Source: Neurology - April 9, 2014 Category: Neurology Authors: Brooks, B., Fischer, M., Sanjak, M., Holsten, S., Kandinov, B., Bockenek, W., Bravver, E., Desai, U., Story, J. S., Pacicco, T., Lindblom, S., Langford, V., Wright, K., Ward, A., Lucas, N., Smith, N., Nichols, M., Lary, C., Nemeth, J., Russo, P. Tags: ALS: Trials and Biomarkers Source Type: research

Stem Cells for Cell-Based Therapies
The world of stem cells We know the human body comprises many cell types (e.g., blood cells, skin cells, cervical cells), but we often forget to appreciate that all of these different cell types arose from a single cell—the fertilized egg. A host of sequential, awe-inspiring events occur between the fertilization of an egg and the formation of a new individual: Embryonic stem (ES) cells are also called totipotent cells. The first steps involve making more cells by simple cell division: one cell becomes two cells; two cells become four cells, etc. Each cell of early development is undifferentiated; that is, it is...
Source: ActionBioscience - December 28, 2012 Category: Science Authors: Ali Hochberg Source Type: news

Agent Orange exposure and disease prevalence in Korean Vietnam veterans: The Korean veterans health study.
In conclusion, Agent Orange exposure increased the prevalence of endocrine disorders, especially in the thyroid and pituitary gland; various neurologic diseases; COPD; and liver cirrhosis. Overall, this study suggests that Agent Orange/2,4-D/TCDD exposure several decades earlier may increase morbidity from various diseases, some of which have rarely been explored in previous epidemiologic studies. PMID: 24906069 [PubMed - as supplied by publisher]
Source: Environmental Research - June 3, 2014 Category: Environmental Health Authors: Yi SW, Hong JS, Ohrr H, Yi JJ Tags: Environ Res Source Type: research

Can Quantitative Muscle Strength and Functional Motor Ability Differentiate the Influence of Age and Corticosteroids in Ambulatory Boys with Duchenne Muscular Dystrophy?
Conclusion The baseline data analysis of this natural history study indicates that the outcomes measures utilized in this study were sensitive to the age related differences in strength and motor function that are characteristic of disease progression boys with DMD; however treatment effects were less likely to be identified. These findings reflect the difficulty inherent in obtaining the statistical power needed to substantiate intervention efficacy in the small, heterogeneous samples sizes that are characteristic of DMD clinical studies. Isokinetic dynamometry revealed variability in the muscles affected, which has been ...
Source: PLOS Currents Muscular Dystrophy - July 8, 2016 Category: Neurology Authors: cbuckon Source Type: research

New treatment for SMA offers hope for Arianna
For the first few months of Arianna Condon’s life, everything was moving along fine. She was a happy baby, and seemed to be developing much like her older sister, Tessa. “She was gaining weight, and seemed to be doing great,” says Arianna’s mom, Marina. “She did have problems with reflux, but it was nothing too unusual for a baby.” But by the time Arianna was 3 months old, Marina started to have concerns. Arianna wasn’t lifting her head the way Tessa had at that age. Something didn’t seem right. “I brought it up to her pediatrician, but she told me that all babies develop differently and there wasn’t a ...
Source: Thrive, Children's Hospital Boston - January 16, 2018 Category: Pediatrics Authors: Ellen Greenlaw Tags: Diseases & Conditions Our Patients’ Stories Dr. Basil Darras spinal muscular atrophy spinal muscular atrophy program Spinraza Source Type: news

Neuroscience is the Next Oncology
by Michael D. Ehlers, MD, PhD Dr. Ehlers is with Biogen in Cambridge, Massachusetts. Innov Clin Neurosci. 2018;15(3–4):15–16 Funding: No funding was received for the preparation of this article. Disclosures: Dr. Ehlers is an employee and shareholder at Biogen Inc. in Cambridge, Massachusetts. Prominent and expensive failures in Alzheimer’s disease therapies have led to a contagious belief system in some parts of the biopharma industry that neuroscience is just too hard, too risky, and too uncertain. But, might this belief system itself be a residual bias of the past? Close inspection reveals all the signs of a coming...
Source: Innovations in Clinical Neuroscience - April 1, 2018 Category: Neuroscience Authors: ICNS Online Editor Tags: Commentary Current Issue Source Type: research

First treatment available for spinal muscular atrophy
NHS England negotiates deal on drug cost Related items fromOnMedica Clinicians need better options if we are to stop over-medication Call for NHS England to ‘think again’ on limiting treatment Green light for primary progressive MS drug Stroke survivors need more help taking medicine Resources wasted on low-risk blood pressure patients
Source: OnMedica Latest News - May 14, 2019 Category: UK Health Source Type: news

Novartis ’ Spinal Drug Gets FDA Approval, $2 Million Price Tag
(CNN) — The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age 2. And then came the price tag: $2.125 million for a one-time treatment. The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis. “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” Dr. Ned Sharpless, the FDA’s acting commissioner, said in a statement Friday. ̶...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - May 24, 2019 Category: Consumer Health News Authors: Health – CBS Boston Tags: Health News CNN Novartis Source Type: news

Never before has it been more exciting and important to be a neurologist
What are the current trends and hot topics in neurology? The increasing insights into the genetics and molecular bases of neurological disorders open new perspectives for specific and personalized treatments. This is evidenced most dramatically by the antisense therapy for spinal muscular atrophy. Promising neurological research currently targets neurodegenerative diseases such as amyotrophic lateral sclerosis, Parkinson’s disease, Duchenne muscular atrophy, or Huntington’s disease. Likewise, brain-machine interfaces and neuroprosthetics offer huge potential. Never before has it been more important and more exciting to...
Source: BioMed Central Blog - December 9, 2019 Category: General Medicine Authors: Victoria Hentschke Tags: Uncategorized Source Type: blogs