Avidity Biosciences Appoints Joseph Baroldi as Chief Operating Officer
SAN DIEGO, Feb. 12, 2020 -- (Healthcare Sales & Marketing Network) -- Avidity Biosciences, Inc. (Avidity), a privately-held biotechnology company pioneering Antibody Oligonucleotide Conjugates (AOCs™), announced today the appointment of Joseph Baroldi as ... Biopharmaceuticals, Personnel Avidity Biosciences, Antibody-Oligonucleotide Conjugates, muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 12, 2020 Category: Pharmaceuticals Source Type: news

'I trust you': How a Lancaster family helped lay the building blocks for gene-therapy breakthroughs at Nationwide Children ’s
Andrew is the “And” in Andelyn Biosciences. He was 3 in 2001 when he was diagnosed with Duchenne muscular dystrophy, the most common and among the most aggressive forms of the inherited condition. Patients usually don’t reach 20. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 30, 2020 Category: Biotechnology Authors: Carrie Ghose Source Type: news

Six patients with rare blood disease are doing well after gene therapy clinical trial
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.“With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant,&rdqu...
Source: UCLA Newsroom: Health Sciences - January 28, 2020 Category: Universities & Medical Training Source Type: news

Gene scissors against incurable muscular disease
(Technical University of Munich (TUM)) Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum M ü nchen) have developed a gene therapy that may provide permanent relief for those suffering from DMD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 27, 2020 Category: International Medicine & Public Health Source Type: news

Heart-function protein may help muscular dystrophy patients live longer
(Rutgers University) Rutgers-led discovery may help prevent muscular dystrophy-related heart disease, the leading cause of death in patients with Duchenne muscular dystrophy (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 14, 2020 Category: Biology Source Type: news

The impact of gun violence on those already dying: perspectives from a palliative care physician - Vente TM.
The consult was for a 19-year-old man with Duchenne muscular dystrophy who was in the ICU with respiratory distress and weight loss. Given his degenerative disease, establishing care with the palliative care team made perfect sense. It was, by all mean... (Source: SafetyLit)
Source: SafetyLit - January 8, 2020 Category: International Medicine & Public Health Tags: Commentary Source Type: news

Biotech firm makes $2.8B deal to license muscular dystrophy gene therapy
A gene therapy invented at Nationwide Children's Hospital just landed a licensing deal worth as much as $2.8 billion for a Boston biotech, and now the company's Columbus, Ohio office will play a big role in getting the treatment to the market. Sarepta Therapeutics Inc. has licensed the commercial rights outside of the United States to the Swiss pharmaceutical giant Roche for $1.1 billion in cash and stock up front. Sarepta also can earn as much as $1.7 billion in milestone payments, plus royalties.… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 30, 2019 Category: Pharmaceuticals Authors: Carrie Ghose Source Type: news

Drug Trials Snapshots: VYONDYS 53
VYONDYS 53 is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). It is to be used only in patients who have a specific mutation of the dystrophin gene. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 26, 2019 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Roche Dives Deeper Into Gene Therapy With $1.15 Bln Sarepta Licensing Deal Roche Dives Deeper Into Gene Therapy With $1.15 Bln Sarepta Licensing Deal
Roche Holding AG made its second major gene therapy deal in a year on Monday, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc's investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States.Reuters Health Information (Source: Medscape Critical Care Headlines)
Source: Medscape Critical Care Headlines - December 24, 2019 Category: Intensive Care Tags: Family Medicine/Primary Care News Source Type: news

Roche dives deeper into gene therapy with $1.15 billion Sarepta licensing deal
Roche Holding AG made its second major gene therapy deal in a year on Monday, spending $1.15 billion to obtain the rights to Sarepta Therapeutics Inc's investigational drug to treat duchenne muscular dystrophy (DMD) outside the United States. (Source: Reuters: Health)
Source: Reuters: Health - December 24, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche enters $1.15 billion licensing deal for Sarepta gene therapy
Roche entered into a $1.15 billion licensing agreement with Sarepta Therapeutics to obtain the right to launch and commercialize Sarepta's investigational gene therapy for Duchenne muscular dystrophy (DMD) outside the United States. (Source: Reuters: Health)
Source: Reuters: Health - December 23, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche enters licensing agreement with Sarepta Therapeutics to improve the lives of patients living with Duchenne muscular dystrophy
Basel, 23 December 2019 - Roche (SIX: RO, ROG; OTCQX: RHHBY) and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), today announced the signing of a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin),   Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the terms of the agreement, Sarepta will receive an upfront payment of $750million in cash and $400million in equity. In addition, Sarepta is eligible to receive regulatory and sales m ilestones, and royalties on net sales. Roche and Sarepta will equally s...
Source: Roche Investor Update - December 23, 2019 Category: Pharmaceuticals Source Type: news

Roche enters licensing agreement with Sarepta Therapeutics to improve the lives of patients living with Duchenne muscular dystrophy
Basel, 23 December 2019 - Roche (SIX: RO, ROG; OTCQX: RHHBY) and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), today announced the signing of a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin),   Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the terms of the agreement, Sarepta will receive an upfront payment of $750million in cash and $400million in equity. In addition, Sarepta is eligible to receive regulatory and sales m ilestones, and royalties on net sales. Roche and Sarepta will equally s...
Source: Roche Media News - December 23, 2019 Category: Pharmaceuticals Source Type: news

Rare diseases: customizing cures, patient by patient
Yale geneticist Monkol Lek, Ph.D., who has muscular dystrophy, wants to cure a rare form of the disease. (Source: Yale Science and Health News)
Source: Yale Science and Health News - December 20, 2019 Category: Universities & Medical Training Source Type: news

In surprise decision, US approves muscular dystrophy drug
The U.S. Food and Drug Administration has approved a second drug to treat muscular dystrophy, the debilitating disease that causes loss of movement and early death (Source: ABC News: Health)
Source: ABC News: Health - December 14, 2019 Category: Consumer Health News Tags: Health Source Type: news

Sarepta shares surge after surprise approval of Duchenne Muscular Dystrophy drug
Shares of Sarepta Therapeutics Inc soared 36% on Friday after U.S. regulators shocked Wall Street by approving the company's treatment for a muscle-wasting disorder less than four months after rejecting the drug over safety concerns. (Source: Reuters: Health)
Source: Reuters: Health - December 13, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA Approves Previously Rejected Muscular Dystrophy Treatment
The drug Vyondys 53 has met a surrogate endpoint of protein production, but there ’s no clear proof it has a clinical benefit. (Source: The Scientist)
Source: The Scientist - December 13, 2019 Category: Science Tags: News & Opinion Source Type: news

FDA OKs Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy FDA OKs Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy
Golodirsen is the first treatment for Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - December 13, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

FDA oks Sarepta muscular dystrophy drug after previous rejection
The U.S. FDA granted an accelerated approval to Sarepta Therapeutics'therapy for Duchenne muscular dystrophy, reversing its original rejection. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - December 13, 2019 Category: Pharmaceuticals Source Type: news

FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation
The U.S. Food and Drug Administration today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is estimated that about 8 percent of patients with DMD have this mutation. (Source: World Pharma News)
Source: World Pharma News - December 13, 2019 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

In an about face, FDA approves Sarepta's 2nd Duchenne drug
The FDA unexpectedly reversed course Thursday, approving the second treatment developed by Sarepta Therapeutics for the rare disease Duchenne muscular dystrophy just a few months after rejecting the drug.   Sarepta (Nasdaq: SRPT) announced Thursday evening that the agency had approved its second drug, called Vyondys 53. After the drug was originally rejected by the FDA in August due to concerns over the risk of infection at the infusion site and renal toxicity, the company filed an appeal. The comp any… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 13, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

U.S. FDA gives early approval to Sarepta's newest DMD treatment
The U.S. Food and Drug Administration on Thursday granted an early approval to Sarepta Therapeutics Inc's second treatment for Duchenne muscular dystrophy (DMD). (Source: Reuters: Health)
Source: Reuters: Health - December 13, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA Okays First Newborn Screening Test for Duchenne MD FDA Okays First Newborn Screening Test for Duchenne MD
The FDA has approved the marketing of the GSP Neonatal Creatine Kinase-MM kit to aid in the screening of newborns for Duchenne muscular dystrophy.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - December 12, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

Today, the @US_FDA authorized the marketing of the first test to aid in newborn screening for Duchenne Muscular Dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Find out more about the new approval https://go.usa.gov/xpdH4   pic.twitter.com/0pFyl2MYOw
Today, the @US_FDA authorized the marketing of the first test to aid in newborn screening for Duchenne Muscular Dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Find out more about the new approval https://go.usa.gov/xpdH4  pic.twitter.com/0pFyl2MYOw (Source: Food and Drug Adminstration (FDA): CDRHNew)
Source: Food and Drug Adminstration (FDA): CDRHNew - December 12, 2019 Category: Medical Devices Authors: ( at FDADeviceInfo) Source Type: news

FDA Approves Vyondys 53 (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53
CAMBRIDGE, Mass., Dec. 12, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Vyondys 53™... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - December 12, 2019 Category: Drugs & Pharmacology Source Type: news

Woman with muscular dystrophy says cosplay 'saved her life'
Amber Guzman, 32, from Long Beach, California, has muscular dystrophy. The incurable condition has left Amber in a wheelchair. (Source: the Mail online | Health)
Source: the Mail online | Health - December 2, 2019 Category: Consumer Health News Source Type: news

UMass Medical School Works To Ease Challenge Of Duchenne Muscular Dystrophy
WORCESTER (CBS) — Caring for a child with Duchenne Muscular Dystrophy can be incredibly challenging, but thanks to a local family’s philanthropy and a local medical school’s commitment, treatment is becoming easier for patients and their families. At just over a year old, Diego Ramirez was diagnosed with Duchenne Muscular Dystrophy, or DMD, a progressive genetic disorder in boys that leads to muscle breakdown. “His muscles felt a little tighter for me, and I felt he was delayed in his development,” said Diego’s mom, Leslie Guzman. Most boys with DMD lose the ability to walk by age 12 and...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - November 29, 2019 Category: Consumer Health News Authors: Health – CBS Boston Tags: Boston News Health Healthwatch Syndicated CBSN Boston Syndicated Local Dr. Mallika Marshall Duchenne muscular dystrophy Source Type: news

JAR 914: Father Declares War on Duchenne Muscular Dystrophy
New Jersey dad declares war on son's incurable disease - Duchenne Muscular Dystrophy. JAR of Hope nonprofit funds start of medical infusions to slow rare disease that effects 20,000 in U.S. (Source: Disabled World)
Source: Disabled World - November 19, 2019 Category: Disability Tags: USA - Americas Source Type: news

Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment. (Source: The Scientist)
Source: The Scientist - November 12, 2019 Category: Science Tags: News & Opinion Source Type: news

Intelligent Metamaterials Could Make MRI ’s Safer and More Affordable
Boston University researchers have developed a new, "intelligent" metamaterial -- which costs less than ten bucks to build -- that could revolutionize MRI, making the entire MRI process faster, safer, and more accessible to patients around the world. The technology, which builds on previous metamaterial work by the team, was described in a new paper in Advanced Materials. MRI is used by clinicians to diagnose medical problems by spotting abnormalities that could indicate anything from a torn meniscus to muscular dystrophy. But MRIs are expensive, expose patients to radiation, and they take a long time -- often th...
Source: MDDI - November 6, 2019 Category: Medical Devices Authors: MDDI Staff Tags: Imaging R & D Source Type: news

Intelligent Metamaterials Could Make MRI ’s Faster and More Affordable
Boston University researchers have developed a new, "intelligent" metamaterial -- which costs less than ten bucks to build -- that could revolutionize MRI, making the entire MRI process faster, safer, and more accessible to patients around the world. The technology, which builds on previous metamaterial work by the team, was described in a new paper in Advanced Materials. MRI is used by clinicians to diagnose medical problems by spotting abnormalities that could indicate anything from a torn meniscus to muscular dystrophy. But MRIs are expensive, and they take a long time -- often the greater part of an hour for ...
Source: MDDI - November 6, 2019 Category: Medical Devices Authors: MDDI Staff Tags: Imaging R & D Source Type: news

Meat, Fish, and Vegetables: New Data on Heart Disease and Stroke Meat, Fish, and Vegetables: New Data on Heart Disease and Stroke
Dr Christoph Diener on interesting new results in stroke, epilepsy, Duchenne muscular dystrophy, and more.Medscape Neurology (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 2, 2019 Category: Neurology Tags: Neurology & Neurosurgery Commentary Source Type: news

Atrium Health joins forces to advance ground-breaking Muscular Dystrophy treatment
A new therapy that could change the face of Muscular Dystrophy treatment has been discovered in Charlotte. This new therapy, developed at the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health was created for the treatment of Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i), and provides a novel approach that targets the disease at its source. Limb Girdle Muscular Dystrophy Type 2i, is one of the rarest forms of this progressive disease. Generally diagnosed in adolescents… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - November 1, 2019 Category: Biotechnology Authors: Atrium Health Source Type: news

A transiton toolkit for Duchenne Muscular Dystrophy
The care of individuals with Duchenne Muscular Dystrophy (DMD) now extends into adulthood. This US guidance looks at transition planning relating to health, education, steps towards working vocations, personal care, accessing the home and community and the importance of relationships with others. It includes a comprehensive checklist of things to be considered, discussed and planned during transition. (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - October 29, 2019 Category: Consumer Health News Source Type: news

Spironolactone Noninferior in Duchenne Muscular Dystrophy
MONDAY, Oct. 28, 2019 -- In a head-to-head study of mineralocorticoid receptor antagonists, spironolactone was found to be noninferior to eplerenone for slowing the progression of heart damage in boys with Duchenne muscular dystrophy (DMD),... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - October 28, 2019 Category: Pharmaceuticals Source Type: news

The Director of the NIH Lays Out His Vision of the Future of Medical Science
Our world has never witnessed a time of greater promise for improving human health. Many of today’s health advances have stemmed from a long arc of discovery that begins with strong, steady support for basic science. In large part because of fundamental research funded by the National Institutes of Health (NIH), which traces its roots to 1887, Americans are living longer, healthier lives. Life expectancy for a baby born in the U.S. has risen from 47 years in 1900 to more than 78 years today. Among the advances that have helped to make this possible are a 70% decline in the U.S. death rate from cardiovascular disease ...
Source: TIME: Science - October 24, 2019 Category: Science Authors: Dr. Francis S. Collins Tags: Uncategorized Healthcare medicine Source Type: news

FSHD Society to Hold Landmark " Voice of the Patient " Meeting
Externally led Patient-Focused Drug Development meeting on facioscapulohumeral muscular dystrophy patients will take place in April of 2020(PRWeb October 23, 2019)Read the full story at https://www.prweb.com/releases/fshd_society_to_hold_landmark_voice_of_the_patient_meeting/prweb16664359.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - October 23, 2019 Category: Pharmaceuticals Source Type: news

Cheaper drug just as effective protecting heart in Duchenne muscular dystrophy
A new clinical trial conducted at The Ohio State University Wexner Medical Center found a cost-effective generic medication works just as well as a more expensive drug in preserving cardiovascular function in boys with Duchenne muscular dystrophy (DMD). Results of this multi-center trial are published in Journal of the American Heart Association. (Source: World Pharma News)
Source: World Pharma News - September 25, 2019 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

Texas university will no longer bred dogs with a muscular wasting disease
Texas A&M University will stop breeding canines onsite to have the genetic disease duchenne muscular dystrophy, which eventually leaves sufferers unable to walk. (Source: the Mail online | Health)
Source: the Mail online | Health - September 18, 2019 Category: Consumer Health News Source Type: news

FSHD Society Announces 2020 International Research Congress and...
The nation’s capital will host the world’s premier conferences on facioscapulohumeral muscular dystrophy for all stakeholders(PRWeb September 10, 2019)Read the full story at https://www.prweb.com/releases/fshd_society_announces_2020_international_research_congress_and_connect_conferences/prweb16555337.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 10, 2019 Category: Pharmaceuticals Source Type: news

FDA Denies Approval for Duchenne Muscular Dystrophy Drug FDA Denies Approval for Duchenne Muscular Dystrophy Drug
The FDA has nixed approval of golodirsen injection for the treatment of Duchenne muscular dystrophy, citing concerns over risk for infusion port infections and possible renal toxicity.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 21, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

FDA declines to approve Sarepta's second Duchenne treatment
Sarepta Therapeutics Inc said on Monday the U.S. Food and Drug Administration declined to approve its newest treatment for Duchenne muscular dystrophy (DMD), citing safety concerns including the risk of infection and kidney toxicity. (Source: Reuters: Health)
Source: Reuters: Health - August 20, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA rejects Sarepta's second Duchenne drug
The FDA has denied Sarepta Therapeutics ’ application for its second Duchenne muscular dystrophy treatment, despite the company producing twice the amount of data behind its controversial first product approval in 2016. Sarepta (Nasdaq: SRPT) had hoped to launch a treatment called golodirsen that would have doubled its reach among youn g boys with the disease. The drug, like Sarepta’s sole commercial treatment, was developed to skip over the faulty gene that prevents young boys with the disease… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 20, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

FDA rejects Sarepta's second Duchenne drug
The FDA has denied Sarepta Therapeutics ’ application for its second Duchenne muscular dystrophy treatment, despite the company producing twice the amount of data behind its controversial first product approval in 2016. Sarepta (Nasdaq: SRPT) had hoped to launch a treatment called golodirsen that would have doubled its reach among youn g boys with the disease. The drug, like Sarepta’s sole commercial treatment, was developed to skip over the faulty gene that prevents young boys with the disease… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 20, 2019 Category: Biotechnology Authors: Allison DeAngelis Source Type: news

FDA declines to approve Sarepta's second Duchenne drug
The U.S. Food and Drug Administration declined to approve Sarepta Therapeutics Inc's newest treatment for Duchenne muscular dystrophy (DMD), a rare muscle-wasting disorder that mainly affects boys, the company said on Monday. (Source: Reuters: Health)
Source: Reuters: Health - August 20, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Patient in Sarepta Gene Therapy Trial Develops Serious Illness Patient in Sarepta Gene Therapy Trial Develops Serious Illness
Shares of Sarepta Therapeutics Inc plunged 12% after a notice from the U.S. health agency showed a patient in an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD) was reported to have developed a serious illness.Reuters Health Information (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 10, 2019 Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news

Sarepta says adverse event report for DMD gene therapy erroneously submitted
Sarepta Therapeutics Inc said on Thursday it was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD). (Source: Reuters: Health)
Source: Reuters: Health - August 9, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Sarepta stock tumbles after Duchenne gene therapy complication
Sarepta Therapeutics stock dropped as much as 13 percent Thursday after news emerged that a child participating in a trial of the company's experimental gene therapy for Duchenne muscular dystrophy experienced a serious health complication. Sarepta (Nasdaq: SRPT) has been testing a treatment for the rare disease that would insert a functioning gene into patients with the disease, who have a non-working copy of the gene. The Cambridge biotech's stock quickly dropped Thursday when it was reported… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 8, 2019 Category: Biotechnology Authors: Allison DeAngelis Source Type: news

Sarepta stock tumbles after Duchenne gene therapy complication
Sarepta Therapeutics stock dropped as much as 13 percent Thursday after news emerged that a child participating in a trial of the company's experimental gene therapy for Duchenne muscular dystrophy experienced a serious health complication. Sarepta (Nasdaq: SRPT) has been testing a treatment for the rare disease that would insert a functioning gene into patients with the disease, who have a non-working copy of the gene. The Cambridge biotech's stock quickly dropped Thursday when it was reported… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 8, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news