Sentences with phrase «dystrophy treatment»
Their discovery has potential implications for muscular dystrophy treatment.
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«New muscular dystrophy treatment shows promise in early study.»
In addition to muscular dystrophy treatment research, similar studies might also be conducted in the future on loss of muscle strength during normal or accelerated aging.
«Gene discovery could lead to muscular dystrophy treatment.»
• Solid Biosciences, a Cambridge, Mass. - based Duchenne muscular dystrophy treatment maker, now says it plans an IPO of 7 million shares priced between $ 18 to $ 19 raising $ 129.5 million.
Sarepta Therapeutics, which won a pioneering Food and Drug Administration approval for its Duchenne muscular dystrophy drug last year, has settled a patent dispute with rival BioMarin over the «exon - skipping» technology at heart of the companies» muscular dystrophy treatments.
Sarepta chief Ed Kaye says that the money raised from its review voucher sale will go into funding R&D for future muscular dystrophy treatments.
Not exact matches
The treatment was approved for the devastating rare disease Duchenne muscular dystrophy but created major divisions within the FDA because it simply hadn't demonstrated a whole lot of efficacy, meaning the pricey therapy is essentially being subjected to a real - world clinical test.
The saga of Sarepta Therapeutics» rare disease drug Exondys 51, the first - ever treatment for the degenerative movement disorder Duchenne muscular dystrophy, has taken yet another turn.
When Sarepta Therapeutics nabbed a pioneering approval for a Duchenne muscular dystrophy (DMD) drug last year, it won more than just bragging rights and a controversial FDA approval: the firm also landed a coveted «priority review voucher,» which can either be sold off to another company or used to slash the review period for a future Sarepta treatment.
Pediatric rehabilitation, spasticity / tone evaluation and treatment, long - term care of chronically impaired children, transition of disabled children to adult services, muscular dystrophy, cerebral palsy, orthotics for gait disorders, pediatric orthotics
Muscular dystrophy is a very complex disorder, and when needed, we include specialized treatment from our colleagues in areas such as Pediatric Orthopaedics, Neurology, Neurosurgery, Physical Therapy, Occupational Therapy and Psychiatry.
«Duchenne muscular dystrophy patients have limited treatment options and a desperate need for effective therapies,» said University of Rochester Medical Center (URMC) neurologist Robert Griggs, M.D., lead author of the study.
New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy.
The new study, called FOR - DMD (Finding the Optimum Regimen of Corticosteroids for Duchenne Muscular Dystrophy), will determine whether daily steroid treatment or an alternative regime is more effective in slowing the disease progression and managing side effects.
«Study could herald new treatment for muscular dystrophy.»
Kumar and Hindi believe their research ultimately will lead to improved treatments for muscle wasting diseases such as muscular dystrophy, ALS, cancer cachexia, diabetes, heart disease and others.
Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy — a type of muscular dystrophy that affects the skeletal muscles — have found that the therapy is long - lasting.
«Although there is no cure for muscular dystrophy, improvements in treatments could help control symptoms to improve quality of life.
«Both of the designed linker proteins may possibly be used in the future as a gene therapy treatment for congenital muscular dystrophy,» says Rüegg.
But questions remained about how important the genetic factors are and whether the disease is a discrete disorder like muscular dystrophy, for example, or a trait requiring treatment only when it reaches a certain threshold.
The treatment is for Stargardt's macular dystrophy, which affects 1 in 8000 people in the US.
Yin's research also has implications for the treatment of other diseases involving muscle damage, including muscular dystrophy.
There is no treatment for FSHD, which is thought by many to be the most common type of muscular dystrophy.
Laboratory fruit flies are used for quick screening of candidate drug treatments for Duchenne muscular dystrophy.
Current treatments for Duchenne muscular dystrophy are limited to steroids and physical therapy that slow disease progression and lessen symptoms.
«Duchenne muscular dystrophy is a stem cell disease: Study paves the way for new treatments for devastating genetic disease.»
«This completely changes our understanding of Duchenne muscular dystrophy and could eventually lead to far more effective treatments.»
«Eteplirsen approved in US for treatment of duchenne muscular dystrophy.»
Oligonucleotide treatments recently have been approved by the Food and Drug Administration for two neuromuscular diseases: Duchenne's muscular dystrophy and spinal muscular atrophy (SMA).
Such an understanding is urgently needed, as no treatments are yet available for muscular dystrophies and muscle - wasting disorders,» stated Alessandra Sacco, Ph.D., associate professor in the Development, Aging, and Regeneration Program at SBP and senior author of the study.
«This could therefore have an impact on the treatment of muscular diseases, including myopathies and muscular dystrophies.»
The discovery could have an important impact on the treatment of muscular diseases such as myopathies and muscular dystrophies.
«This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy.»
A preclinical study led by researchers in the United States has found that a new oral drug shows early promise for the treatment of muscular dystrophy.
The study authors previously found out that NF - κB is active in dystrophin - deficient muscle years before the onset of symptoms, suggesting that very early treatment of Duchenne Muscular Dystrophy patients with VBP15 may prevent or delay the onset of some clinical symptoms.
Studying such disease - resistant people could point to new treatments for rare conditions like Duchenne muscular dystrophy.
LONDON — Wellcome Trust, the United Kingdom's largest biomedical research charity, today announced more than # 4 million in support for a pioneering, and potentially controversial, IVF treatment that could prevent some forms of muscular dystrophy and other diseases caused by defective mitochondria, the energy - generating organelle in cells.
The team previously found that NF - κB is active in dystrophin - deficient muscle years before the onset of symptoms, suggesting that very early treatment of Duchenne muscular dystrophy patients with VBP15 may prevent or delay the onset of some clinical symptoms.
Johns Hopkins University biologists have found that a protein that plays a key role in the lives of stem cells can bolster the growth of damaged muscle tissue, a step that could potentially contribute to treatments for muscle degeneration caused by old age and diseases such as muscular dystrophy.
The finding provides new insight into how diseases, such as muscular dystrophy, that disconnect brain and body occur, as it points to novel treatment targets, said neuroscientist Dr. Lin Mei.
Doctors in the US have already begun their first tests of genetically manipulated cells in patients with cancer; comparable treatments for immune deficiency or Duchenne muscular dystrophy are much further off.
Discovered a major mechanism of action for glucocorticoid hormones in skeletal muscle that have important implications for treatment of muscular dystrophy
What's more, Spiegelman's team plans to investigate the potential of irisin to advance the treatment of diseases such as muscular dystrophy and muscle wasting.
Advanced Cell Technology (ACT), based in Marlborough, Mass., will begin testing its retinal cell treatment this year in a dozen patients with Stargardt's macular dystrophy, an inherited degenerative eye disease that leads to blindness in children.
Part 2 of this two - part article reviews lessons from Spark Therapeutics» pivotal program for Luxturna, a gene therapy approved for the treatment of patients with retinal dystrophy associated with confirmed biallelic mutation in the RPE65 gene, and summarize key considerations for the clinical development and commercialization of gene therapies.
July 13, 2016 Simple procedure could improve treatment for common eye disease: Rejuvenating the cornea could reduce the need for cornea transplants A new, minimally invasive procedure appears to be effective for many patients with Fuchs endothelial dystrophy (FED), a common eye disease, without the potential side effects and cost of the current standard of care, a cornea transplant.
More and more scientists are using the powerful new gene - editing tool known as CRISPR / Cas9, a technology isolated from bacteria, that holds promise for new treatment of such genetic diseases as cystic fibrosis, muscular dystrophy...
If the new approach to financing research hastens the discovery of a treatment, «it can serve as a model for other hereditary forms of neurodegenerative diseases like muscular dystrophy and Huntington's disease,» he added.
Presentations included: Genetics Primer & Clinical Updates by Angelika Erwin, MD, PhD, Expanded Carrier Screening — What you Need to Know by Amy Shealy, MS, LGC, Recent Advances in the Treatment and Management of Cystic Fibrosis by Silvia Cardenas, MD, Advances in the Management of Duchenne Muscular Dystrophy by Neil Freidman, MBChB, Autism Genetics — PTEN and beyond by Thomas W. Frazier, PhD, Thoracic Aorta Aneurysm and Dissection by Apostolos «Paul» Psychogios, MD, FACMG, Update on Clinical Breast Cancer Genetics by Holly Pederson, MD, Colon Cancer by Brandie Leach, MS, LGC and The Role of Biomarkers in Current Diagnosis of Alzheimer's Disease by Jagan Pillai, MD, PhD.