New CRISPR technique skips over portions of genes that can cause disease
(University of Illinois at Urbana-Champaign) In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell's internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. Such targeted editing could one day be useful for treating genetic diseases caused by mutations in the genome, such as Duchenne's muscular dystrophy, Huntington's disease or some cancers. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 16, 2018 Category: International Medicine & Public Health Source Type: news

This matrix delivers healing stem cells to injured elderly muscles
(Georgia Institute of Technology) Muscles of the elderly and of patients with Duchene muscular dystrophy have trouble regenerating. A new nanohydrogel with muscle stem cells has boosted muscle growth in mouse models while protecting the stem cells from immune reactions that usually weaken or destroy them. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 15, 2018 Category: International Medicine & Public Health Source Type: news

A protein could be key to preserving heart function in Duchenne muscular dystrophy
(American Heart Association) The brain-derived neurotrophic factor protein, known to be vital for brain function, might hold the key for preserving heart function in children and young adults with Duchenne muscular dystrophy.Heart failure often kills Duchenne patients early in life and there is no effective treatment.Developing therapies designed to activate or supplement brain-derived neurotrophic factor could give new hope to patients at risk for Duchenne cardiomyopathy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 1, 2018 Category: International Medicine & Public Health Source Type: news

Understanding muscular dystrophy and home remedies to help manage it
(Natural News) Muscular dystrophy is a group of genetic diseases wherein the skeletal muscles weaken and break down over time. This condition is brought on by the absence of dystrophin, an essential protein that plays an important role in normal muscle function. People who lack dystrophin will often have difficulty swallowing and moving, and will... (Source: NaturalNews.com)
Source: NaturalNews.com - July 28, 2018 Category: Consumer Health News Source Type: news

Annual Expedition Mt. Kinabalu Raises Awareness of a Duchenne Clinical...
Enrollment for Duchenne Muscular Dystrophy Clinical Trial Now Underway(PRWeb July 25, 2018)Read the full story at https://www.prweb.com/releases/annual_expedition_mt_kinabalu_raises_awareness_of_a_duchenne_clinical_trial_bringing_treatments_to_boys_in_malaysia/prweb15650024.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - July 25, 2018 Category: Pharmaceuticals Source Type: news

Cambridge biotech Summit pivots from failed trial, layoffs with $4.5M from CARB-X
CARB-X's $4.5 million award comes just a few weeks after the company shut down on a Duchenne muscular dystrophy trial and laid off most of their employees in Cambridge. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - July 11, 2018 Category: American Health Authors: Allison DeAngelis Source Type: news

Cambridge biotech Summit pivots from failed trial, layoffs with $4.5M from CARB-X
CARB-X's $4.5 million award comes just a few weeks after the company shut down on a Duchenne muscular dystrophy trial and laid off most of their employees in Cambridge. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 11, 2018 Category: Biotechnology Authors: Allison DeAngelis Source Type: news

Researchers regenerate muscle cells in mice with muscular dystrophy
Refined cells from a type of benign tumor regenerated skeletal muscle stem cells in mice with muscular dystrophy, according to a new study. (Source: Health News - UPI.com)
Source: Health News - UPI.com - July 5, 2018 Category: Consumer Health News Source Type: news

Some existing anti-cancer drugs may act in part by targeting RNA
Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells. The research offers another approach for tackling diseases that have been considered "undruggable," including amyotrophic lateral sclerosis (ALS), muscular dystrophy, cystic fibrosis and certain cancers. (Source: World Pharma News)
Source: World Pharma News - June 29, 2018 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

CBT Improves Function, Reduces Fatigue in Muscular Dystrophy CBT Improves Function, Reduces Fatigue in Muscular Dystrophy
A new study shows cognitive-behavioral therapy with optional exercise is associated with reductions in fatigue and increased activity levels, but increased falls are a concern.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 28, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news

Dietary supplement increases muscle force by 50% percentin the Duchenne muscular dystrophy mouse model
(Universit é Laval) A dietary supplement derived from glucose increases muscle-force production in the Duchenne muscular dystrophy (DMD) mouse model by 50 percent in ten days, according to a study conducted by researchers from Universit é Laval's Faculty of Medicine and Centre hospitalier universitaire (CHU) de Qu é bec Research Centre-Universit é Laval. These results pave the way for a clinical study to test the treatment's effectiveness on humans. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 28, 2018 Category: International Medicine & Public Health Source Type: news

Some existing anti-cancer drugs may act in part by targeting RNA, study shows
(Scripps Research Institute) The research offers another approach for tackling diseases that have been considered 'undruggable,' including amyotrophic lateral sclerosis (ALS), muscular dystrophy, cystic fibrosis and certain cancers. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 28, 2018 Category: International Medicine & Public Health Source Type: news

Summit plunges 80 percent after muscle-wasting disorder drug fails
(Reuters) - Britain's Summit Therapeutics Plc said on Wednesday that it would stop developing its Duchenne muscular dystrophy (DMD) drug after it failed a mid-stage study, wiping out nearly 80 percent of its market value. (Source: Reuters: Health)
Source: Reuters: Health - June 27, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Summit plunges 80 pct after muscle-wasting disorder drug fails
(Reuters) - Britain's Summit Therapeutics Plc said on Wednesday that it would stop developing its Duchenne muscular dystrophy (DMD) drug after it failed a mid-stage study, wiping out nearly 80 percent of its market value. (Source: Reuters: Health)
Source: Reuters: Health - June 27, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Summit plunges 70 percent as muscle-wasting disorder drug fails
(Reuters) - Britain's Summit Therapeutics Plc said on Wednesday that it would stop developing its Duchenne muscular dystrophy (DMD) drug, ezutromid, after it failed a mid-stage study, sending its shares plunging more than 70 percent. (Source: Reuters: Health)
Source: Reuters: Health - June 27, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Curing a deadly childhood disease, sharing her love of science, and a sleek ’68 Corvette drive this biochemist
Spend a brief amount of time with biochemist Rachelle Crosbie-Watson and you ’ll quickly realize that “drive” is one of her favorite words.With equal enthusiasm, she ’ll describe studying “the small molecules that drive life,” and her 1968 convertible Corvette being “a blast to drive.” The symmetry is hard to miss: Crosbie-Watson drives a classic muscle car to UCLA, where she studies the biochemical reactions that drive muscle cell functions. Her lab is hotly pursuing new drugs that one day may halt the progression of a deadly childhood muscle-wasting disease, allowing k...
Source: UCLA Newsroom: Health Sciences - June 26, 2018 Category: Universities & Medical Training Source Type: news

Johnson & Johnson hits the Big Apple with latest JLabs site
Johnson & Johnson (NYSE:JNJ) opened its latest life science incubator in New York City, the healthcare giant said today. The 30,000-square-foot JLabs @ NYC is a collaboration between Johnson & Johnson Innovation, New York State and the New York Genome Center. Sited at the genome center in SoHo, the incubator is home to 26 startups and has room for four more, New Brunswick, N.J.-based J&J said. “Johnson & Johnson has deep entrepreneurial roots in New York and we are pleased to see our unique JLabs model applied in this rich ecosystem to foster the creation of new healthcare innovations that have t...
Source: Mass Device - June 21, 2018 Category: Medical Devices Authors: Brad Perriello Tags: Funding Roundup Research & Development johnsonandjohnson Source Type: news

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