Severe Lipodystrophy Following Prolonged Administration of Antisense Oligonucleotides

This case report describes a severe adverse event following antisense oligonucleotide treatment for muscular dystrophy that occurred in 6 pediatric patients.
Source: JAMA Dermatology - Category: Dermatology Source Type: research

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Viravuth (“Voot”) Yin, associate professor of regenerative biology and medicine at MDI Biological Laboratory and chief scientific officer at Novo Biosciences, Inc., in Bar Harbor, Maine. Credit: MDI Biological Laboratory. In 1980, a week after his 6th birthday, Viravuth (“Voot”) Yin immigrated with his mother, grandfather, and three siblings from Cambodia to the United States. Everything they owned fit into a single, 18-inch carry-on bag. They had to build new lives from almost nothing. So, it’s perhaps fitting that Yin studies regeneration, the fascinating ability of some animals, such ...
Source: Biomedical Beat Blog - National Institute of General Medical Sciences - Category: Research Authors: Tags: Being a Scientist Cool Creatures Regeneration Research Organisms RNA Source Type: blogs
A new study has found 'never before reported' cells in the spines of people with ALS. The discovery may lead to a better understanding of the condition.
Source: Health News from Medical News Today - Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news
ABSTRACT Duchenne de Boulogne is known mainly by the disease eponymously named “ Duchenne muscular dystrophy ” , or pseudohypertrophic muscular dystrophy, although some experts consider that the original description of this disease does not belong to him. Less well known are the facts related to the tragic death of his beloved wife shortly after the birth of his only child, the unjustifiable distance he was forced to keep from this son for more than 30 years, and of being humiliated and professionally despised by his peers. These events made the life of this physician, physiologist, researcher and inventor extr...
Source: Arquivos de Neuro-Psiquiatria - Category: Neurology Source Type: research
ABSTRACT Duchenne de Boulogne is known mainly by the disease eponymously named “ Duchenne muscular dystrophy ” , or pseudohypertrophic muscular dystrophy, although some experts consider that the original description of this disease does not belong to him. Less well known are the facts related to the tragic death of his beloved wife shortly after the birth of his only child, the unjustifiable distance he was forced to keep from this son for more than 30 years, and of being humiliated and professionally despised by his peers. These events made the life of this physician, physiologist, researcher and inventor extr...
Source: Arquivos de Neuro-Psiquiatria - Category: Neurology Source Type: research
ABSTRACT Duchenne de Boulogne is known mainly by the disease eponymously named “ Duchenne muscular dystrophy ” , or pseudohypertrophic muscular dystrophy, although some experts consider that the original description of this disease does not belong to him. Less well known are the facts related to the tragic death of his beloved wife shortly after the birth of his only child, the unjustifiable distance he was forced to keep from this son for more than 30 years, and of being humiliated and professionally despised by his peers. These events made the life of this physician, physiologist, researcher and inventor extr...
Source: Arquivos de Neuro-Psiquiatria - Category: Neurology Source Type: research
Twenty six participants representing patients, funding agencies and basic and clinical scientists involved in research into skeletal muscle stem cells and muscular dystrophies from France, Germany, Italy, India, UK, Australia, Spain, USA, The Netherlands and Switzerland met in Hoofddorp, The Netherlands on 25 – 27 January 2019. The meeting was held under the auspices of the ENMC and ENMC main sponsors, with the additional support of Muscular Dystrophy UK.
Source: Neuromuscular Disorders - Category: Neurology Authors: Tags: Workshop report Source Type: research
Conditions:   Bethlem Myopathy;   Ullrich Congenital Muscular Dystrophy 1, Digenic, Col6A1/Col6A2;   Ullrich Congenital Muscular Dystrophy 1, Autosomal Recessive;   Ullrich Congenital Muscular Dystrophy 1, Autosomal Dominant;   Bethlem Myopathy 1, Autosomal Recessive;   UCMD;   BTHLM1 Intervention:   Other: Patient Registry Sponsor:   Newcastle-upon-Tyne Hospitals NHS Trust Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Researchers at Tufts University and the Chinese Academy of Sciences have developed a new lipid nanoparticle which can deliver CRISPR/Cas9 gene editing tools into organs with high efficiency, suggesting that the system is promising for clinical applic...
Source: Medgadget - Category: Medical Devices Authors: Tags: Genetics Source Type: blogs
Muscle weakness is a defining characteristic of Muscular Dystrophy (MD); however, yet while speculated, objective measures of muscle weakness has not been reported in relation to quality of life in adults with...
Source: Health and Quality of Life Outcomes - Category: International Medicine & Public Health Authors: Tags: Research Source Type: research
Conclusion These qualitative data from chronic NPPV users suggest that both M-NPPV and nasal BPAP may interfere with speaking but that speech is usually better and speaking is usually easier with M-NPPV. These findings can be explained primarily by the nature of the 2 ventilator delivery systems and their interfaces. PMID: 31306604 [PubMed - in process]
Source: American Journal of Speech-Language Pathology - Category: Speech-Language Pathology Authors: Tags: Am J Speech Lang Pathol Source Type: research
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