Yale neuroscientist awarded grant to investigate ALS
Junjie Guo, assistant professor of neuroscience, has been awarded a grant of $297,678 from the Muscular Dystrophy Association. (Source: Yale Science and Health News)
Source: Yale Science and Health News - May 14, 2019 Category: Universities & Medical Training Source Type: news

Boosting muscle stem cells to treat muscular dystrophy and aging muscles
(Sanford Burnham Prebys Medical Discovery Institute) Scientists from Sanford Burnham Prebys have uncovered a molecular signaling pathway involving Stat3 and Fam3a proteins that regulates how muscle stem cells decide whether to self-renew or differentiate -- an insight that could lead to muscle-boosting therapeutics for muscular dystrophies or age-related muscle decline. The study was published in Nature Communications. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - April 17, 2019 Category: International Medicine & Public Health Source Type: news

Novo Biosciences achieves major milestones in moving trodusquemine into clinical trials
(Novo Biosciences) Novo Biosciences Inc., has achieved several major milestones in its mission of bringing its breakthrough drug candidate, trodusquemine (aka MSI-1436), to market as a potential regenerative medicine treatment for heart disease and Duchenne muscular dystrophy (DMD). Trodusquemine is a repurposed drug candidate that has already been shown to be well tolerated by patients. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 12, 2019 Category: International Medicine & Public Health Source Type: news

Small molecule targets cause of adult onset muscular dystrophy
Researchers designed a small molecule that, in mice, blocks the mutated RNA responsible for adult onset muscular dystrophy. The findings suggest a new avenue to develop therapeutics. (Source: NIH Research Matters from the National Institutes of Health (NIH))
Source: NIH Research Matters from the National Institutes of Health (NIH) - April 9, 2019 Category: Consumer Health News Source Type: news

Ex-Sarepta execs join new Atlas-backed muscular disease startup fueled by $50M
A new Atlas Venture-founded startup launched Wednesday, backed by $50 million, to develop drugs for a currently untreated form of muscular dystrophy and other muscle-based diseases. Dyne Therapeutics emerged from stealth mode this week with Series A funding from Dutch firm Forbion and Cambridge firms MPM Capital and Atlas. Dyne was founded and incubated by Atlas over the past year. Its first focus area is myotonic dystrophy type 1, a rare genetic disease that causes muscle weakness and affects… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - April 3, 2019 Category: American Health Authors: Allison DeAngelis Source Type: news

Sarepta plans second Duchenne drug application this year
With one drug already under FDA consideration, Sarepta Therapeutics Inc. is now planning to submit another Duchenne muscular dystrophy drug for the federal agency's approval this year. Sarepta (Nasdaq: SRPT) is the market leader in treatments for Duchenne muscular dystrophy, a rare genetic disease that causes young boys muscles to deteriorate. It developed the first treatment specifically for the disorder, Exondys 51, which brought in $301 million in sales last year. The Cambri dge biotech could… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 28, 2019 Category: Pharmaceuticals Source Type: news

​For many boys with Duchenne Muscular Dystrophy, bright hope lies just beyond reach
Even for the parents of a child with muscular dystrophy whose child qualifies for a treatment that may stop the disease, good news may be followed by agonizing, life-or-death choices. What treatments seem most promising? Should he be enrolled in a trial with a placebo arm? (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - March 26, 2019 Category: Health Management Authors: Gina Kolata Source Type: news

​For many boys with Duchenne Muscular Dystrophy, bright hope lies just beyond reach
Even for the parents of a child with muscular dystrophy whose child qualifies for a treatment that may stop the disease, good news may be followed by agonizing, life-or-death choices. What treatments seem most promising? Should he be enrolled in a trial with a placebo arm? (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 26, 2019 Category: Biotechnology Authors: Gina Kolata Source Type: news

ASGCT To Honor Four Ground-Breaking Researchers at 22nd Annual Meeting
Drs. John Rossi, Steven Gray, Anna Kajaste-Rudnitski and the Muscular Dystrophy Association will be recognized for their tremendous contributions to gene and cell therapy at the American Society of...(PRWeb March 26, 2019)Read the full story at https://www.prweb.com/releases/asgct_to_honor_four_ground_breaking_researchers_at_22nd_annual_meeting/prweb16194040.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 26, 2019 Category: Pharmaceuticals Source Type: news

For Many Boys With Duchenne Muscular Dystrophy, Bright Hope Lies Just Beyond Reach
Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify. (Source: NYT Health)
Source: NYT Health - March 25, 2019 Category: Consumer Health News Authors: GINA KOLATA Tags: Muscular Dystrophy Drugs (Pharmaceuticals) Disabilities Clinical Trials Muscles Muscular Dystrophy Assn Men and Boys Source Type: news

Image of the Day: Dystrophin Restored
CRISPRed heart muscle cells from humans gain the ability to make a protein missing in Duchenne muscular dystrophy. (Source: The Scientist)
Source: The Scientist - March 21, 2019 Category: Science Tags: Image of the Day Source Type: news

Risk factors for falls among boys under 18 years with muscular dystrophy - Hong Y, Wang Y, Zhang Y, Royer JA, Cai B, Mann JR, McDermott S.
PURPOSE: Studies have shown that children with muscular dystrophy are at increased risk for falls, however there is insufficient information about what predicts the first and subsequent events. The purpose of this study was to describe the experience of in... (Source: SafetyLit)
Source: SafetyLit - March 21, 2019 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news

Scientists find method to boost CRISPR efficiency
(UT Southwestern Medical Center) Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 6, 2019 Category: International Medicine & Public Health Source Type: news

Medical News Today: Pollutants may speed up ALS progression
New research suggests that environmental pollution, in general, and pesticides, in particular, may advance disease progression in people with ALS. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - March 2, 2019 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Medical News Today: ALS: New technique prevents toxic protein deposits in cells
Clumps of faulty TDP-43 protein inside nerve cells are common in ALS and other neurological diseases. Could this new technique offer a way to prevent them? (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - February 27, 2019 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Grant Marks Two Decades of NIH Support for Muscular Dystrophy Research
The University of Rochester Medical Center has received $8 million from the National Institutes of Health to support pioneering research on muscular dystrophy. The grant, which is a renewal of URMC ’s Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, will fund ongoing work to investigate the genetic mechanisms and progression of this complex multi-system disease, research that has led scientists to the threshold of potential new therapies for myotonic dystrophy. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - February 25, 2019 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news

New drug for Duchenne muscular dystrophy clears phase 1 clinical trial
Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects. The investigational drug edasalonexent, an oral NF-κB inhibitor, has the potential to slow the progression of the disease for all patients with DMD. (Source: World Pharma News)
Source: World Pharma News - February 21, 2019 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

New drug for Duchenne muscular dystrophy clears phase 1 clinical trial testing in boys
(IOS Press) Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 21, 2019 Category: International Medicine & Public Health Source Type: news

Single CRISPR treatment provides long-term benefits in mice
(Duke University) Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 18, 2019 Category: International Medicine & Public Health Source Type: news

Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53
CAMBRIDGE, Mass., Feb. 14, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced the Food and Drug Administration, Division of Neurology (the Division), has accepted... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - February 14, 2019 Category: Drugs & Pharmacology Source Type: news

UMN study provides new insight into use of cell replacement therapies to treat muscular dystrophies
(University of Minnesota Medical School) The University of Minnesota Medical School continues its legacy of advancing cell replacement therapies with a scientific breakthrough that highlights the promise of cell therapies for muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 13, 2019 Category: International Medicine & Public Health Source Type: news

URMC Designated as a Duchenne Care Center
Parent Project Muscular Dystrophy, a nonprofit organization dedicated to advancing care and research for patients with Duchenne muscular dystrophy, has named the University of Rochester Medical Center a Certified Duchenne Care Center. This program of leading centers, created in 2014 by PPMD, reviews and recognizes clinics nationwide for their outstanding neuromuscular programs. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - February 12, 2019 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news

New research insights hold promise for kids with DMD
(Children's National Health System) Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects. Eplerenone, a heart failure drug, is used in older patients to treat cardiomyopathy, a leading cause of mortality for people with DMD. A new medicine under development appears to combine the beneficial effects of these drugs for the heart and muscle while also showing improved safety in experimental models. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 11, 2019 Category: International Medicine & Public Health Source Type: news

Discovery points to innovative new way to treat Duchenne muscular dystrophy
(The Ottawa Hospital) Researchers at The Ottawa Hospital and the University of Ottawa have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. As reported in Cell Stem Cell, the team restored muscle stem cell function that is impaired in Duchenne muscular dystrophy, resulting in efficient regeneration of the muscle and preventing the progressive loss of muscle strength characteristic of the disease. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - January 31, 2019 Category: International Medicine & Public Health Source Type: news

Medical News Today: ALS: A new therapy may be in sight
New research, led by Harvard scientists, identifies a novel potential therapeutic target for treating amyotrophic lateral sclerosis (ALS). (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - January 17, 2019 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Muscle stem cells can drive cancer that arises in Duchenne muscular dystrophy
People with Duchenne muscular dystrophy (DMD) can develop an otherwise-rare muscle cancer, called rhabdomyosarcoma, due to the muscle cells' continuous work to rebuild the damaged tissue. However, little is known about how the cancer arises, hindering development of a treatment or test that could predict cancer risk. (Source: World Pharma News)
Source: World Pharma News - January 15, 2019 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

Muscle stem cells can drive cancer that arises in Duchenne muscular dystrophy
(Sanford Burnham Prebys Medical Discovery Institute) Scientists from Sanford Burnham Prebys Medical Discovery Institute (SBP) have demonstrated that muscle stem cells may give rise to rhabdomyosarcoma that occurs during DMD--and identified two genes linked to the tumor's growth. The research, performed using a mouse model of severe DMD, helps scientists better understand how rhabdomyosarcoma develops in DMD--and indicates that ongoing efforts to develop treatments that stimulate muscle stem cells should consider potential cancer risk. The study was published in Cell Reports on January 15, 2019. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - January 15, 2019 Category: Cancer & Oncology Source Type: news

Researchers overcome hurdle in CRISPR gene editing for muscular dystrophy
(University of Missouri-Columbia) The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions. A research team led by Dongsheng Duan, PhD, at the University of Missouri School of Medicine has identified and overcome a barrier in CRISPR gene editing that may lay the foundation for sustained treatments using the technique. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 8, 2019 Category: International Medicine & Public Health Source Type: news

Home Monitoring of Lung Function Feasible in Teens With Duchenne
MONDAY, Jan. 7, 2019 -- In adolescent patients with Duchenne muscular dystrophy (DMD), use of a handheld device (HHD) to assess pulmonary function at home is feasible and provides reliable data, according to an analysis recently published in the... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - January 7, 2019 Category: Pharmaceuticals Source Type: news

An Experimental Procedure Could Help More Families Have Healthy Babies. But It ’s Not Allowed in the U.S.
When Noah Shulman was born a few days after Christmas 2016, his parents Kristelle and Evan had no reason to worry about him. The pregnancy went smoothly, and so did the birth. But within a few days of taking his first breath, Noah began to struggle. He wasn’t feeding, so he started losing weight. He was also lethargic. Several pediatricians reassured the Shulmans that they were probably just overly sensitive to Noah’s symptoms because Kristelle is a nurse and Evan is a physician assistant–a case of first-time-parent-white-coat syndrome. “They kind of dismissed us as neurotic parents,” says Eva...
Source: TIME: Science - January 3, 2019 Category: Science Authors: Alice Park Tags: Uncategorized fertility Research Source Type: news

An Experimental Procedure Could Help More Families Have Healthy Babies. But It ’s Not Allowed in the U.S.
When Noah Shulman was born a few days after Christmas 2016, his parents Kristelle and Evan had no reason to worry about him. The pregnancy went smoothly, and so did the birth. But within a few days of taking his first breath, Noah began to struggle. He wasn’t feeding, so he started losing weight. He was also lethargic. Several pediatricians reassured the Shulmans that they were probably just overly sensitive to Noah’s symptoms because Kristelle is a nurse and Evan is a physician assistant–a case of first-time-parent-white-coat syndrome. “They kind of dismissed us as neurotic parents,” says Eva...
Source: TIME: Health - January 3, 2019 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized fertility Research Source Type: news

U.S. Drug Developer Capricor Places Duchenne Drug Trial on Hold U.S. Drug Developer Capricor Places Duchenne Drug Trial on Hold
Capricor Therapeutics Inc said on Wednesday it had put on hold a clinical trial of its drug to treat Duchenne muscular dystrophy (DMD), a muscle-wasting disorder, citing a safety review.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 28, 2018 Category: Consumer Health News Tags: Family Medicine/Primary Care News Source Type: news

Company halts drug trial for rare disease
Capricor Therapeutics has announced that it is stopping progress on a trial for a new Duchenne muscular dystrophy treatment. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - December 27, 2018 Category: Pharmaceuticals Source Type: news

U.S. drug developer Capricor places Duchenne drug trial on hold
Capricor Therapeutics Inc said on Wednesday it had put on hold a clinical trial of its drug to treat Duchenne muscular dystrophy (DMD), a muscle-wasting disorder, citing a safety review. (Source: Reuters: Health)
Source: Reuters: Health - December 26, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Capricor places Duchenne drug trial on hold
Capricor Therapeutics Inc said on Wednesday it had put on hold a clinical trial of its drug to treat Duchenne muscular dystrophy, a muscle-wasting disorder. (Source: Reuters: Health)
Source: Reuters: Health - December 26, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053) in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53
CAMBRIDGE, Mass., Dec. 20, 2018 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA)... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - December 20, 2018 Category: Drugs & Pharmacology Source Type: news

Monitoring lung function at home in teens with Duchenne muscular dystrophy
(IOS Press) Heart and lung complications are responsible for much of the morbidity and mortality associated with Duchenne muscular dystrophy (DMD). Although regular monitoring of pulmonary function is recommended in order to detect deterioration, compliance with routine testing, such as hospital-based spirometry, is frequently poor. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 17, 2018 Category: International Medicine & Public Health Source Type: news

New mouse model may speed identification of promising muscular dystrophy therapies
(Massachusetts General Hospital) A Massachusetts General Hospital research team has created a new mouse model of a common form of muscular dystrophy with the potential of rapidly distinguishing promising therapeutic drugs from those unlikely to be successful. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 12, 2018 Category: International Medicine & Public Health Source Type: news

UMN researchers give new insight to muscular dystrophy patients
(University of Minnesota Medical School) New research by University of Minnesota scientists has revealed the three dimensional structure of the DUX4 protein, which is responsible for the disease, facioscapulohumeral muscular dystrophy (FSHD). Unlike the majority of genetic diseases, FSHD is not caused by a protein that is missing or not functioning properly. Rather it is caused when a functioning, normal, protein shows up in a place where it doesn't belong (in muscles). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 12, 2018 Category: International Medicine & Public Health Source Type: news

Newly published model of FSHD and a potential gene therapy to improve functional outcomes
(Nationwide Children's Hospital) Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent dominantly inherited muscular dystrophy in the world. To date, there are no pharmacologic treatments available for the more than 850,000 people affected worldwide. Developing models to use for testing potential therapeutics has been a challenge for the research community. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 16, 2018 Category: International Medicine & Public Health Source Type: news

Improved Mobility With Metformin in Myotonic Dystrophy Type 1 Improved Mobility With Metformin in Myotonic Dystrophy Type 1
This Phase II RCT showed promise for metformin for improving the mobility of patients with myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.Brain (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - November 14, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news

Moths and magnets could save lives
(Rice University) Rice University bioengineers have combined a virus that infects moths with magnetic nanoparticles to create a potential new therapy for inherited genetic diseases like muscular dystrophy, sickle cell, cystic fibrosis, spinal muscular atrophy and some forms of cancer. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - November 13, 2018 Category: Cancer & Oncology Source Type: news

Life-changing intersection between big data and therapeutic pipeline in neuromuscular disease
(MWW Group) A new report funded by the Muscular Dystrophy Association (MDA) and released by the IQVIA Institute for Human Data Science indicates that genetic testing and breakthrough therapies will transform the diagnosis and care of neuromuscular disease within the next decade. The report, 'Understanding Neuromuscular Disease Care,' highlights current gaps in care and opportunities to optimize care and accelerate the emergence of new therapies. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 30, 2018 Category: International Medicine & Public Health Source Type: news

Sarepta CEO charts path forward on Duchenne drug amid stock slide
Sarepta has seen its share price decline in the wake of data from a small study of its gene therapy drug for Duchenne muscular dystrophy. But the Cambridge biotech is moving forward with its clinical trial work and expansion plans. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 11, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

Glucose-derived dietary supplement increases muscle-force production in Duchenne muscular dystrophy
(Natural News) According to a study, a dietary supplement extracted from glucose can help boost muscle-force production. Researchers involved in the study believe this can one day help patients with Duchenne muscular dystrophy (DMD). The study, which involved researchers from Université Laval’s Faculty of Medicine and Centre hospitalier universitaire (CHU) de Québec Research Centre-Université Laval, was published in the... (Source: NaturalNews.com)
Source: NaturalNews.com - October 8, 2018 Category: Consumer Health News Source Type: news

Medical News Today: How can gold help repair muscle injuries?
In a new and innovative study, researchers have found that gold nanoparticles attached to anti-inflammatory agents can work to promote muscle regeneration. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - October 5, 2018 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational "hotspot" in the human DMD gene. We used adeno-associated viruses to deliver CRISPR gene editing components to four dogs and examined dystrophin protein expression 6 weeks after intramuscular delivery (n = 2) or 8 weeks after systemic delivery (n = 2). After systemic delivery in skeletal muscle, dystrophin was restored to levels ranging from 3 to 90% of normal, depending on muscle type. In cardi...
Source: ScienceNOW - October 4, 2018 Category: Science Authors: Amoasii, L., Hildyard, J. C. W., Li, H., Sanchez-Ortiz, E., Mireault, A., Caballero, D., Harron, R., Stathopoulou, T.-R., Massey, C., Shelton, J. M., Bassel-Duby, R., Piercy, R. J., Olson, E. N. Tags: Medicine, Diseases, Molecular Biology reports Source Type: news

Gene editing and muscular dystrophy
(Source: ScienceNOW)
Source: ScienceNOW - October 4, 2018 Category: Science Authors: Kiberstis, P. A. Tags: Medicine, Diseases, Molecular Biology twis Source Type: news

Sarepta Gene Therapy Appears To Help Fourth Boy With Duchenne Muscular Dystrophy
“The cautionary note is this all has to be confirmed in a larger trial,” says Douglas Ingram, Sarepta’s president and chief executive. “Our goal is to bring this therapy rapidly to patients around the world,” Ingram says. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - October 3, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Source Type: news

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See something of interest? Please share our postings with colleagues in your institutions! Spotlight September is National Preparedness Month. Disasters Happen. Prepare Now. Learn How. This week: Learn Life Saving Skills. Member Highlights: Let us shine a spotlight on the amazing work you do! NNLM MAR is always interested in learning about health outreach projects and activities that are happening in Pennsylvania, New York, New Jersey and Delaware. Share your story with us to receive a Member Highlight on the MARquee! National Network of Libraries of Medicine News Funding Available: NNLM MAR has funding available for ...
Source: NN/LM Middle Atlantic Region Blog - September 14, 2018 Category: Databases & Libraries Authors: Hannah Sinemus Tags: Weekly Postings Source Type: news