Sarepta shares soar as Duchenne gene therapy shows promise
(Reuters) - Sarepta Therapeutics' shares jumped 60 percent on Tuesday after promising results from a gene therapy study positioned the specialty drugmaker as a potential leader in the market for Duchenne muscular dystrophy (DMD) treatments. (Source: Reuters: Health)
Source: Reuters: Health - June 19, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Cambridge biotech Sarepta tops $11B market cap on gene therapy data
Sarepta Therapeutics on Tuesday released promising data from a small, early-stage trial of its experimental gene therapy treatment for Duchenne muscular dystrophy, showing that the drug significantly boosted an important muscle protein in three boys with the disease. Shares of Sarepta (Nasdaq: SRPT) soared Tuesday morning on the news, propelling its market cap from $6.9 billion to more than $11 billion briefly, before dropping back to $10.6 billion as of 11:15 a.m. The stock increase of 54 percent… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 19, 2018 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

Sarepta shares soar as gene therapy study shows promise
(Reuters) - Sarepta Therapeutics Inc shares jumped 60 percent on Tuesday after the drugmaker reported promising results from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD). (Source: Reuters: Health)
Source: Reuters: Health - June 19, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Sarepta stock soars as early-stage gene therapy data shows promise
(Reuters) - Sarepta Therapeutics Inc reported promising results on Tuesday from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD), driving the company's shares up 60 percent. (Source: Reuters: Health)
Source: Reuters: Health - June 19, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

A Gene Therapy Appears ToReplaceMissing ProteinIn Muscular Dystrophy Patients
An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, according to results being presented by the drugmaker Sarepta Therapeutics at an investor event. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - June 19, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:SLDB NASDAQ:SRPT NYSE:PFE Source Type: news

Upgrading the toolbox for Duchenne muscular dystrophy research with a new rabbit model
(The Company of Biologists) Research to improve our understanding of Duchenne muscular dystrophy (DMD), and the development of new therapies, has previously relied on mouse models. However, physiological differences between the two species has limited how successfully findings in mice can be applied to humans. A newly developed rabbit model, created through the use of CRISPR/Cas-9 genome editing, exhibits greater clinical similarity to human patients than the mouse models currently in use, with huge potential to advance DMD research. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - June 4, 2018 Category: Biology Source Type: news

FSH Society Announces Third Annual World FSHD Day to be Celebrated on...
Advocates, patient and families from around the world to unite to raise awareness for facioscapulohumeral muscular dystrophy(PRWeb June 04, 2018)Read the full story at https://www.prweb.com/releases/2018/06/prweb15528649.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - June 4, 2018 Category: Pharmaceuticals Source Type: news

CHMP Advises Against Approval for Eteplirsen in DMD CHMP Advises Against Approval for Eteplirsen in DMD
The EMA's Committee for Medicinal Products for Human Use recommends against approval of eteplirsen for Duchenne muscular dystrophy but extends approval of ataluren to younger patients with the disease.International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 1, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery News Alert Source Type: news

Medical News Today: ALS: Immune cells may slow disease progression
For the first time in humans, researchers administered immune T cells to patients with ALS. This slowed down the progression of the disease. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - May 24, 2018 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Duchenne Muscular Dystrophy: New Resource to Guide Clinical Care Duchenne Muscular Dystrophy: New Resource to Guide Clinical Care
Comprehensive new resources guide clinicians in all settings to provide optimal care to patients with Duchenne muscular dystrophy.CDC Expert Commentary (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - May 21, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery Commentary Source Type: news

What is BACM?
Discussion Leg pain in children has a broad differential diagnosis and includes more innocuous problems such as growing pains and transient synovitis but also more serious problems including neurological diseases or malignancy. Occult or self-limited trauma are also common causes. A review can be found here. Limping can be painful or painless and has its own differential diagnosis which can overlap with leg pain. A differential diagnosis for limping can be found here. Muscle weakness also has its’ own differential diagnosis which can be found here. Learning Point Benign acute childhood myositis (BACM) has other names...
Source: PediatricEducation.org - May 21, 2018 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Myonexus Therapeutics receives FDA Rare Pediatric Disease Designation
(CincyTech) Myonexus Therapeutics, a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children's Hospital announced today that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for the MYO-101 program, an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (LGMD) type 2E. In February 2018, the MYO-101 program was granted Orphan Drug Designation (ODD) by FDA. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - May 16, 2018 Category: Biology Source Type: news

Much policy on newborn blood spot screening ignores evidence
Many national recommendations on screening are not based on evidence of key benefits and harms, say researchers Related items fromOnMedica Experts advise against screening for Duchenne Muscular Dystrophy All babies in Europe should be screened for heart defects at birth The next generation of prenatal testing: let ’s proceed with caution Antenatal flu drugs not linked to newborn risks (Source: OnMedica Latest News)
Source: OnMedica Latest News - May 10, 2018 Category: UK Health Source Type: news

Students create exoskeleton app for patient with muscular dystrophy
(Michigan State University) A team of Michigan State University students invented an exoskeleton app to help a patient suffering from muscular dystrophy. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - May 3, 2018 Category: International Medicine & Public Health Source Type: news

Medical News Today: ALS: Most physically active have '26 percent higher risk'
A large study conducted in three countries provides good evidence of a link between a history of vigorous physical activity and the risk of developing ALS. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - April 24, 2018 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy
Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular dystrophy (DMD). The first boy received an infusion of the mini-dystrophin gene on March 22nd, administered under the supervision of principal investigator, Edward Smith, MD, Associate Professor of Pediatrics and Neurology at Duke University Medical Center. (Source: World Pharma News)
Source: World Pharma News - April 23, 2018 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news

Myonexus Therapeutics receives FDA orphan drug designation for LGMD type 2E treatment
(CincyTech) Myonexus Therapeutics , a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children's Hospital announce the US Food and Drug Administration (FDA) has granted Orphan Drug designation to Myonexus' lead candidate, MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 23, 2018 Category: International Medicine & Public Health Source Type: news

Muscular dystrophy doesn't stop Adam living life to the full
Adam has a rare muscle-wasting disorder, but he isn't letting that stop him enjoying life. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - April 19, 2018 Category: Consumer Health News Source Type: news

Medical News Today: ALS: How 'toxic' proteins could protect neurons
Researchers have found that large, fibrous aggregates thought to drive Lou Gehrig's disease might actually help to protect motor neurons. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - April 17, 2018 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

ALS: How 'toxic' proteins could protect neurons
Researchers have found that large, fibrous aggregates thought to drive Lou Gehrig's disease might actually help to protect motor neurons. (Source: Parkinson's Disease News From Medical News Today)
Source: Parkinson's Disease News From Medical News Today - April 17, 2018 Category: Neurology Tags: Muscular Dystrophy / ALS Source Type: news

Pfizer launches Duchenne gene therapy study, joining Sarepta and Solid
Pfizer has officially entered the race to cure Duchenne muscular dystrophy with gene therapy, joining Cambridge-based Sarepta Therapeutics and Solid Biosciences as the only companies to use the experimental technology on a boy with the muscle-wasting disease. Pfizer (NYSE: PFE) announced on Thursday that the first patient in a small, early-stage trial of its gene therapy drug had been dosed on March 22. Earlier this year, both Sarepta (Nasdaq: SRPT) and Solid Biosciences (Nasdaq: SLDB) began their… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 12, 2018 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

UCLA research could be first step toward healing the hearts of children with Duchenne
After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.Now, researchers at theDavid Geffen School of Medicine at UCLA are pursuing a cutting-edge way to stop heart disease in patients with Duchenne, which affects one in 5,000 male babies born in the United States. Their work, which is supported by a David Geffen School of Medicine Seed Grant, is just one of a number of projects underway at the medical school in which interdisciplinary groups of UCLA researchers are p...
Source: UCLA Newsroom: Health Sciences - April 6, 2018 Category: Universities & Medical Training Source Type: news

Boy with muscle-wasting illness uses holographs to see grandparents  
EXCLUSIVE: Harrison Smith, 11, with terminal muscular dystrophy has used Microsoft's HoloLens headset and the world's first HoloPortal to talk to his grandfather. (Source: the Mail online | Health)
Source: the Mail online | Health - March 30, 2018 Category: Consumer Health News Source Type: news

Investors sue Solid Biosciences, say they were duped about Duchenne drug ’s safety
Cambridge-based Solid Biosciences is facing legal claims from investors after disclosing safety issues with its lead drug, a gene therapy treatment for Duchenne muscular dystrophy. Solid (Nasdaq: SLDB) revealed on March 14 that the FDA had halted an early-stage study of the drug after one patient was hospitalized. While Solid said that the patient had recovered, the disclosure sent shares of the newly-public biotech tumbling more than 60 percent. The FDA setback caught the attention of a handful… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - March 28, 2018 Category: Health Management Authors: Max Stendahl Source Type: news

Investors sue Solid Biosciences, say they were duped about Duchenne drug ’s safety
Cambridge-based Solid Biosciences is facing legal claims from investors after disclosing safety issues with its lead drug, a gene therapy treatment for Duchenne muscular dystrophy. Solid (Nasdaq: SLDB) revealed on March 14 that the FDA had halted an early-stage study of the drug after one patient was hospitalized. While Solid said that the patient had recovered, the disclosure sent shares of the newly-public biotech tumbling more than 60 percent. The FDA setback caught the attention of a handful… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 28, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

Tamoxifen and raloxifene slow down the progression of muscular dystrophy
(Elsevier) Steroids are currently the only available treatment to reduce the repetitive cycles of inflammation and disease progression associated with functional deterioration in patients with muscular dystrophy (MD). A study reported in The American Journal of Pathology showed that a new treatment approach using the selective estrogen receptor modulators (SERMs) tamoxifen and raloxifene significantly improved cardiac, respiratory, and skeletal muscle functions and increased bone density in both male and female mice with the same gene defects as a subset of patients with MD. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - March 20, 2018 Category: Biology Source Type: news

Human 'chimeric' cells restore crucial protein in Duchenne muscular dystrophy
(University of Illinois at Chicago) Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy -- a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 16, 2018 Category: International Medicine & Public Health Source Type: news

Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable
UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.The research waspublished in the journal ACS Nano by senior author Paul Weiss, UC Presidential Chair and distinguished professor of chemistry and biochemistry, materials science and engineering, and member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.Gene the...
Source: UCLA Newsroom: Health Sciences - March 15, 2018 Category: Universities & Medical Training Source Type: news

Shares of Solid Biosciences crater as FDA halts Duchenne gene therapy trial
Less than two months after pulling off one of the biggest initial public offerings for a local biotech in recent years, Cambridge-based Solid Biosciences said that the FDA had halted a trial of its gene therapy treatment for Duchenne muscular dystrophy, sending shares of the company into a tailspin. Solid (Nasdaq: SLDB) said Wednesday that the FDA had stopped an early-stage study of the drug after one patient was hospitalized due to reduced platelet and red blood cell count. The company added that… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - March 14, 2018 Category: American Health Authors: Max Stendahl Source Type: news

Shares of Solid Biosciences crater as FDA halts Duchenne gene therapy trial
Less than two months after pulling off one of the biggest initial public offerings for a local biotech in recent years, Cambridge-based Solid Biosciences said that the FDA had halted a trial of its gene therapy treatment for Duchenne muscular dystrophy, sending shares of the company into a tailspin. Solid (Nasdaq: SLDB) said Wednesday that the FDA had stopped an early-stage study of the drug after one patient was hospitalized due to reduced platelet and red blood cell count. The company added that… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 14, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

House Rejects GOP Bill Giving Terminally Ill Patients the ‘Right to Try’ Experimental Treatment
(WASHINGTON) — The House rejected legislation Tuesday easing how experimental drugs are provided to people with terminal illnesses, as Democrats calling the bill risky and misleading overcame support from President Donald Trump and emotional arguments by Republican lawmakers and ailing constituents. The vote for the measure was 259-140, but that fell short of the two-thirds majority the GOP needed to prevail under special procedures. Since the Senate approved similar legislation last August, Republicans could revisit the legislation under rules that would require only a simple majority for passage, perhaps after rewo...
Source: TIME: Health - March 14, 2018 Category: Consumer Health News Authors: Alan Fram / AP Tags: Uncategorized APH Congress healthytime onetime Source Type: news

Sarepta Says FDA Welcomes Review Of New Muscular Dystrophy Drug
Sarepta Therapeutics says regulators at the Food and Drug Administration have told it they were open to reviewing its next experimental treatment for Duchenne muscular dystrophy, meaning that it could file an application for the new medicine by the end of this year. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - March 12, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:SRPT Source Type: news

Sarepta Therapeutics Announces Plan to Submit a New Drug Application (NDA) for Accelerated Approval of Golodirsen (SRP-4053) in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53
CAMBRIDGE, Mass., March 12, 2018 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases,... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - March 12, 2018 Category: Drugs & Pharmacology Source Type: news

Patients living longer with Duchenne muscular dystrophy pose new challenge for caregivers
(Case Western Reserve University) Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of independence, personal relationships, emotional health, and intimacy. To address these shifting circumstances, as well as reflect promising new treatment options, new guidelines aimed at physicians who care for DMD patients have recently been issued. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 12, 2018 Category: International Medicine & Public Health Source Type: news

Medical News Today: Diesel exhaust may raise risk of neurological disease
A new study has revealed that men with occupations involving exposure to diesel exhaust could be at greater risk of developing ALS, also known as Lou Gehrig's disease. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - February 28, 2018 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Therapy for muscular dystrophy-caused heart failure also improves muscle function in mice
(Cedars-Sinai Medical Center) Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows. The study, published today in Stem Cell Reports, showed that when researchers injected cardiosphere-derived cells (CDCs) into the hearts of laboratory mice with muscular dystrophy, heart function improved along with a marked increase in exercise capacity. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 22, 2018 Category: International Medicine & Public Health Source Type: news

Sarepta shares plunge on U.K. trial halt, but analyst downplays safety fears
Sarepta Therapeutics confirmed Friday that U.K. regulators have temporarily halted a late-stage trial of two drugs for the muscle-wasting diseases Duchenne muscular dystrophy after one patient experienced a serious medical issue. But one analyst downplayed the incident as an “isolated event.” In a statement, Cambridge-based Sarepta (Nasdaq: SRPT) said the patient’s medical issue “could possibly be related” to the blinded study, in which patients were given one of two drugs, called golodirsen… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 9, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

Device that measures cell strength could help identify drugs for asthma, hypertension and muscular dystrophy
Engineers, doctors and scientists at UCLA and Rutgers University have developed a tool that measures the physical strength of individual cells 100 times faster than current technologies.The new device could make it easier and faster to test and evaluate new drugs for diseases associated with abnormal levels of cell strength, including hypertension, asthma and muscular dystrophy. It could also open new avenues for biological research into cell force. It is the first high-throughput tool that can measure the strength of thousands of individual cells at a time.“Our tool tracks how much force individual cells exert over ...
Source: UCLA Newsroom: Health Sciences - February 9, 2018 Category: Universities & Medical Training Source Type: news

New CRISPR method efficiently corrects DMD defect in heart tissue
(UT Southwestern Medical Center) Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 6, 2018 Category: International Medicine & Public Health Source Type: news

Analysis of global duchenne muscular dystrophy patients registry underscores
(IOS Press) Duchenne muscular dystrophy (DMD) is a a rare and fatal genetically-inherited degenerative neuromuscular disease that affects one in 5,000 newborn boys. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 1, 2018 Category: International Medicine & Public Health Source Type: news

Myoediting for Duchenne muscular dystrophy
(Source: ScienceNOW)
Source: ScienceNOW - February 1, 2018 Category: Science Authors: Shilatifard, A. Tags: twis Source Type: news

This company is the result of Nationwide Children's reviving gene therapy in the U.S.
It ’s the stuff of science fiction – replacing defective genes in the human body with new ones that will stop a debilitating disease. But it seems to becoming reality at Michael Triplett’s new Columbus gene therapy company, which is having success controlling a rare muscular dystrophy disease tha t cripples its patients. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - January 29, 2018 Category: Health Management Source Type: news

Two area biotechs raise $210M in IPOs, then watch shares soar
Cambridge-based Solid Biosciences and Boston-based resTORbio raised a combined $210 million in initial public offerings over the past day, and as of 3 p.m. Friday both the company ’s shares were trading significantly up from the IPO price. Solid Biosciences, a company working on drugs to treat Duchenne muscular dystrophy, raised $125 million by pricing its shares at $16, below the $18 to $19 range first proposed. The reason appears to be a disclosure Thursday morning in a federal filing that the… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 26, 2018 Category: Biotechnology Authors: Don Seiffert Source Type: news

Gene editing – and what it really means to rewrite the code of life
We now have a precise way to correct, replace or even delete faulty DNA.Ian Sample explains the science, the risks and what the future may holdSo what is gene editing?Scientists liken it to the find and replace feature used to correct misspellings in documents written on a computer. Instead of fixing words, gene editing rewrites DNA, the biological code that makes up the instruction manuals of living organisms. With gene editing, researchers can disable target genes,correct harmful mutations, and change the activity of specific genes in plants and animals, including humans.What ’s the point?Much of the excitement aro...
Source: Guardian Unlimited Science - January 15, 2018 Category: Science Authors: Ian Sample Science editor Tags: Genetics Medical research Science Health Biology Source Type: news

Study reveals reversibility of Friedreich ’s ataxia in mice
Friedreich ’s ataxia is an inherited disease that causes damage to the nervous system and a loss of coordination that typically progresses to muscle weakness. It can begin causing symptoms in childhood or early adulthood and, over time, it can also lead to vision loss and diabetes.Scientists seeking a better understanding of the disease have tried for years to replicate the disease ’s symptoms and progression in laboratory mice, but until recently have been largely unsuccessful.Now, a team of UCLA researchers has recreated aspects of Friedreich ’s ataxia in mice and shown that many early symptoms of the d...
Source: UCLA Newsroom: Health Sciences - January 2, 2018 Category: Universities & Medical Training Source Type: news

UCLA researchers create skeletal muscle from stem cells
UCLA scientists have developed a new strategy to efficiently isolate, mature and transplant skeletal muscle cells created from human pluripotent stem cells, which can produce all cell types of the body. The findings are a major step toward  the development of a stem cell replacement therapy for muscle diseases including Duchenne muscular dystrophy, which affects approximately 1 in 5,000 boys in the U.S. and is the most common fatal childhood genetic disease.The study, which was published in the journal Nature Cell Biology, was led by senior author April Pyle, associate professor of microbiology, immunology and molecul...
Source: UCLA Newsroom: Health Sciences - December 18, 2017 Category: Universities & Medical Training Source Type: news

New 'checkpoint' model that could identify potential drugs to treat genetic disorders
(University of Bradford) A new 'checkpoint' model which can be used to identify potential treatments for genetic disorders such as cystic fibrosis and Duchenne muscular dystrophy (DMD) has been proposed by a team of Bradford scientists. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 18, 2017 Category: International Medicine & Public Health Source Type: news

UCLA researchers create skeletal muscle from stem cells
(University of California - Los Angeles Health Sciences) UCLA scientists have developed a new strategy to efficiently isolate, mature and transplant skeletal muscle cells created from human pluripotent stem cells, which can produce all cell types of the body. The findings are a major step towards developing a stem cell replacement therapy for muscle diseases including Duchenne Muscular Dystrophy, which affects approximately 1 in 5,000 boys in the US and is the most common fatal childhood genetic disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 18, 2017 Category: International Medicine & Public Health Source Type: news

Study: The medical community isn’t prepared for the looming pandemic of neurodegenerative diseases like Parkinson’s
(Natural News) Parkinson’s disease is the second most common neurodegenerative disease plaguing people today, with an estimated 1 million Americans are battling this illness – more than Lou Gehrig’s disease, muscular dystrophy and multiple sclerosis combined. Parkinson’s causes certain nerve cells in the brain to die, affecting movement and causing worsening tremors and stiffness. The... (Source: NaturalNews.com)
Source: NaturalNews.com - December 13, 2017 Category: Consumer Health News Source Type: news

Nationwide Children's gene therapy spinout raises $2.5M to test muscular dystrophy treatment
A gene therapy startup that spun out of Nationwide Children's Hospital six months ago has raised $2.5 million toward testing a treatment for a form of muscular dystrophy in patients. Myonexus Therapeutics Inc. is the fourth company to come out of gene therapy research at the Columbus hospital and the only one located in Central Ohio. CEO Michael Triplett and COO Bruce Halpryn are based in New Albany, and all of the contract research it sponsors takes place at Children's. “ The clinical impact… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 13, 2017 Category: Pharmaceuticals Authors: Carrie Ghose Source Type: news