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Duchenne muscular dystrophy sufferers cannot walk
Brothers Elliot Johnson, 14, and Henry, 11, from Pennsylvania fear they won't be able to walk again after the FDA pulled the drugs that have kept their rare muscle disorder under control. (Source: the Mail online | Health)
Source: the Mail online | Health - November 17, 2017 Category: Consumer Health News Source Type: news

Health insurer Anthem eases restrictions on Sarepta's Duchenne treatment
NEW YORK (Reuters) - Health insurer Anthem Inc has eased access to Sarepta Therapeutics' treatment for Duchenne muscular dystrophy, and on Thursday changed its reimbursement policy on its website to "medically necessary." (Source: Reuters: Health)
Source: Reuters: Health - November 9, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Virtual gene editing startup to lease Cambridge lab after $40M round
The company, Exonics Therapeutics, said the Series A round will allow it to hire scientists to conduct preclinical research into a potential treatment for Duchenne muscular dystrophy. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - November 8, 2017 Category: American Health Authors: Max Stendahl Source Type: news

All babies in Europe should be screened for heart defects at birth
Only a few countries, including Poland, Ireland and Switzerland, currently recommend universal screening with pulse oximetry Related items fromOnMedica Blood test identifies high-risk lupus pregnancies Newborns screened for four additional genetic conditions Over 600,000 carry ‘sudden death’ gene fault The next generation of prenatal testing: let ’s proceed with caution Experts advise against screening for Duchenne Muscular Dystrophy (Source: OnMedica Latest News)
Source: OnMedica Latest News - November 8, 2017 Category: UK Health Source Type: news

SLU researcher draws bulls eye around muscular dystrophy drug targets
(Saint Louis University) Scientist Francis M. Sverdrup, Ph.D., studies an inherited type of muscular dystrophy that typically begins with weakness in the face and shoulders before spreading to all skeletal muscles. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 7, 2017 Category: International Medicine & Public Health Source Type: news

Sarepta jumps into the CRISPR fray with Duke gene-editing pact
Cambridge-based Sarepta Therapeutics said Tuesday that it will seek to develop drugs for Duchenne muscular dystrophy using CRISPR/Cas9 gene-editing technology, jumping into an increasingly competitive but still uncertain field. Sarepta (Nasdaq: SRPT) said it had reached an agreement to exclusively license technology related to CRISPR/Cas9 from Duke University. Researchers there, led by Dr. Charles Gersbach, have shown that the technology can be used in mice to remove mutated parts of genes, or exons,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 31, 2017 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

FDA declines to approve PTC's Duchenne drug
(Reuters) - The U.S. Food and Drug Administration has declined to approve PTC Therapeutics Inc's experimental drug to treat Duchenne muscular dystrophy, saying an additional clinical trial would be needed to prove the drug works. (Source: Reuters: Health)
Source: Reuters: Health - October 25, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

FDA declines to approve PTC's Duchenne Muscular Dystrophy drug
(Reuters) - The U.S. Food and Drug Administration has declined to approve PTC Therapeutics Inc's experimental drug to treat Duchenne muscular dystrophy, saying an additional clinical trial would be needed to prove the drug works. (Source: Reuters: Health)
Source: Reuters: Health - October 25, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

FDA rejects muscular dystrophy drug, says it doesn't work
U.S. regulators reject experimental drug for Duchenne muscular dystrophy. (Source: ABC News: Health)
Source: ABC News: Health - October 25, 2017 Category: Consumer Health News Tags: Health Source Type: news

Mom-and-pop drug developers: How these parents are funding a drug for their sons
Both of their sons have a version of muscular dystrophy that has no approved treatment and is slowly robbing the boys of control over their hip and shoulder muscles. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 18, 2017 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

C-Path and CDISC announce therapeutic area user guide for duchenne muscular dystrophy
(Critical Path Institute (C-Path)) C-Path and CDISC announce the availability of TAUG-DMD v1.0: a therapeutic area user guide that describes the most common clinical concepts relevant to Duchenne studies using the CDISC standard format, which allows datasets from different sources to be compared or combined for analysis. Data covered include genetic information, cardiac and respiratory data, as well as results from specific measurements of strength and function. Data standards are also being developed for a variety of functional assessments. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 18, 2017 Category: International Medicine & Public Health Source Type: news

Chronic inflammation may improve drug delivery in MD patients
Researchers at Children's National Health System found chronic inflammation is vital in sustained delivery of a new treatment for Duchenne muscular dystrophy. (Source: Health News - UPI.com)
Source: Health News - UPI.com - October 16, 2017 Category: Consumer Health News Source Type: news

Chronic inflammation may improve drug delivery in muscular dystrophy patients
Researchers at Children's National Health System found chronic inflammation is vital in sustained delivery of a new treatment for Duchenne muscular dystrophy. (Source: Health News - UPI.com)
Source: Health News - UPI.com - October 16, 2017 Category: Consumer Health News Source Type: news

What's the next step for exon skipping therapies to treat duchenne muscular dystrophy?
(Mary Ann Liebert, Inc./Genetic Engineering News) A team of leading European clinicians and scientists presents a unique perspective on how to move forward in the development of exon skipping therapies to treat the severe muscle-wasting disease Duchenne Muscular Dystrophy (DMD). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 16, 2017 Category: International Medicine & Public Health Source Type: news

Capricor Therapeutics Presents Positive Six-Month Results in Duchenne Muscular Dystrophy at World Muscle Society International Congress
Data Set the Stage for the Upcoming HOPE-2 Clinical Trial of CAP-1002 SAINT MALO, France, Oct. 4, 2017 -- (Healthcare Sales & Marketing Network) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), in its presentation today at the 22nd Annual International C... Regenerative Medicine Capricor Therapeutics, Duchenne muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - October 4, 2017 Category: Pharmaceuticals Source Type: news

Armed with fresh trial data, Catabasis doubles down on Duchenne drug
Regrouping after a trial setback involving its lead drug for Duchenne muscular dystrophy, Cambridge-based Catabasis Pharmaceuticals is forging ahead with a late-stage study, touting new data on Wednesday hinting that the treatment can improve the lives of boys with the muscle-wasting disease. Catabasis (Nasdaq: CATB) previously announced in January that a Phase 2 study of the drug, called edasalonexent, had failed to meet its primary goal — significantly reducing leg muscle inflammation compared… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - October 4, 2017 Category: American Health Authors: Max Stendahl Source Type: news

'CRISPR-Gold' fixes Duchenne muscular dystrophy mutation in mice
(University of California - Berkeley) Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 3, 2017 Category: International Medicine & Public Health Source Type: news

Nonviral CRISPR Delivery a Success
Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy. (Source: The Scientist)
Source: The Scientist - October 2, 2017 Category: Science Tags: Daily News Source Type: news

PTC Therapeutics Duchenne drug may work, data inconclusive: FDA panel
(Reuters) - PTC Therapeutics Inc's experimental drug to treat Duchenne muscular dystrophy, a devastating degenerative disease that mostly affects young boys, may work but the company will need to do more work to prove it, an advisory panel to the U.S. Food and Drug Administration concluded on Thursday. (Source: Reuters: Health)
Source: Reuters: Health - September 28, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

FDA pours cold water on PTC Therapeutics' Duchenne drug application
(Reuters) - PTC Therapeutics Inc has not supplied "persuasive" evidence that its experimental drug to treat a form of Duchenne muscular dystrophy is effective, a preliminary review by scientists at the U.S. Food and Drug Administration concluded. (Source: Reuters: Health)
Source: Reuters: Health - September 26, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Phrixus to study P-188 NF in Phase II trial to treat DMD
US-based Phrixus Pharmaceuticals is set to conduct a Phase II clinical trial of Poloxamer 188 NF (P-188 NF) for the treatment of non-ambulatory patients with Duchenne muscular dystrophy (DMD). (Source: Drug Development Technology)
Source: Drug Development Technology - September 17, 2017 Category: Pharmaceuticals Source Type: news

Santhera shares plummet after negative CHMP opinion
ZURICH (Reuters) - Swiss drugmaker Santhera's shares plunged nearly 60 percent in early trading on Friday, after a European panel recommended against approving one of its drugs to be used in patients with Duchenne muscular dystrophy (DMD). (Source: Reuters: Health)
Source: Reuters: Health - September 15, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

University of Minnesota researchers replicate FSH muscular dystrophy in mice
(University of Minnesota) A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease -- which has no approved treatment -- affects an estimated 38,000 Americans and causes muscle degeneration. Scientists inserted into mice a gene called DUX4, which is believed to cause FSHD in humans. When they activated the gene in mice skeletal muscle cells, the animals developed a muscular dystrophy with key features of FSHD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 15, 2017 Category: International Medicine & Public Health Source Type: news

This Is How Telethons Became a Fundraising Tradition
When Hollywood bands together for Hand In Hand: A Benefit For Hurricane Harvey Relief on Tuesday night, the entertainers who show up will be participating in a long-running tradition of lending star power to important causes via televised fundraisers. While the comedian Jerry Lewis became the biggest celebrity telethon-er when he started hosting fundraisers for the Muscular Dystrophy Association in the ’50s he wasn’t the first to do one. Comedian Milton Berle is credited with hosting the first telethon, which NBC broadcasted from 12 p.m. on April 9, 1949, to 3:55 a.m. on April 10th. The 16-hour telecast raised ...
Source: TIME.com: Top Science and Health Stories - September 12, 2017 Category: Consumer Health News Authors: Olivia B. Waxman Tags: Uncategorized health photography Television Source Type: news

​Why Sarepta’s latest trial data may presage another FDA controversy
One year after scoring one of the most controversial FDA approvals in recent history, Cambridge-based Sarepta Therapeutics may again find itself at the center of a debate over the agency ’s standards with a new experimental drug. Sarepta (Nasdaq: SRPT) on Wednesday unveiled data from a small, early-stage trial of one of its potential treatments for Duchenne muscular dystrophy, a genetic disorder in which the absence of a protein called dystrophin causes progressive muscle weaknes s. According to… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - September 6, 2017 Category: Health Management Authors: Max Stendahl Source Type: news

​Why Sarepta’s latest trial data may presage another FDA controversy
One year after scoring one of the most controversial FDA approvals in recent history, Cambridge-based Sarepta Therapeutics may again find itself at the center of a debate over the agency ’s standards with a new experimental drug. Sarepta (Nasdaq: SRPT) on Wednesday unveiled data from a small, early-stage trial of one of its potential treatments for Duchenne muscular dystrophy, a genetic disorder in which the absence of a protein called dystrophin causes progressive muscle weaknes s. According to… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 6, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news

New understanding of how muscles work
(McGill University) Muscle malfunctions may be as simple as a slight strain after exercise or as serious as heart failure and muscular dystrophy. A new technique developed at McGill University now makes it possible to look much more closely at how sarcomeres, the basic building blocks within all skeletal and cardiac muscles, work together. It's a discovery that should advance research into a wide range of muscle malfunctions. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 23, 2017 Category: International Medicine & Public Health Source Type: news

The King of Comedy, Jerry Lewis Dies at 91 - AARP
5 hours ago ... Comedian Jerry Lewis, known for his unique brand of comedy and his work with the Muscular Dystrophy Association, passed away in Las  ... (Source: AARP.org News)
Source: AARP.org News - August 22, 2017 Category: American Health Source Type: news

Duchenne Action Month Needs YOU!
This September, we are asking you, our community, to participate in Duchenne Action Month in order to create awareness, raise money, and make an impact in the fight to #endDuchenne. Check out 10 simple and powerful actions you can take to make an impact this September: www.parentprojectmd.org/actionmonth (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 22, 2017 Category: Neurology Source Type: news

[Webinar] Gene Therapy for Duchenne - August 2017
Last week, PPMD hosted a webinar focused on understanding the different therapies that are being developed that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9, how are they similar and different, and what are the challenges and limitations for each of them. If you missed it, the recording of the webinar is now available! COMING SOON: This fall PPMD will be hosting additional webinars with companies and institutions who are developing gene therapies and/or CRISPR/Cas9 for Duchenne. Stay tuned for more details! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 22, 2017 Category: Neurology Source Type: news

How You Can Participate in the Ataluren Ad Comm
The FDA has announced the scheduling of a September 28th Advisory Committee Meeting for PTC ’s therapy ataluren. Whether submitting written testimony and/or attending the Ad Comm in person, there are multiple ways for Duchenne community members in the U.S. and abroad to have an impact and share your personal experience with the Advisory Committee reviewing this product. Visit our blog to learn how you can participate. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 22, 2017 Category: Neurology Source Type: news

Comic and Telethon Host Jerry Lewis Dies At 91
The legendary actor and comedian helped raise more than $2 billion for muscular dystrophy. (Source: WebMD Health)
Source: WebMD Health - August 21, 2017 Category: Consumer Health News Source Type: news

PPMD's Bone Health Workshop Published
Bone health has long been a confusing and often neglected aspect of Duchenne care. There is very little knowledge of underlying bone health in Duchenne, which is further complicated by the possible effects of glucocorticoids on both bone development and bone density/strength. We applaud the incredible work of this group of experts who participated in PPMD's Bone Health Workshop, and hope that, with increased awareness, further work to prevent osteoporosis and enhance bone health in people living with Duchenne will continue. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 18, 2017 Category: Neurology Source Type: news

PPMD's Every Single [One] Tour: Charleston, WV
Registration for the next stop on PPMD's Every Single [One] Tour is now open! Combining each of the pillars that make up PPMD's mission -- To #EndDuchenne -- the Every Single [One] Tour brings free day-long meetings featuring updates on research, advocacy, and care to the Duchenne community across the country. We hope to see you in Charleston, WV on September 9! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 17, 2017 Category: Neurology Source Type: news

Understanding Clinical Trials in Duchenne
Did you know that there are over 50 clinical trials going on in the US focused on Duchenne& Becker? While having so many clinical trials is exciting, it can also be overwhelming when you are trying to make a decision about if you want to be in a study and if so, which study is the best fit for you and your family. Learn more about the current clinical trial landscape& the PPMD resources that can help you explore clinical trial options. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 7, 2017 Category: Neurology Source Type: news

Eloxx Pharmaceuticals Secures US$8 Million Investment from LSP, Increasing Total Raised in Series C to US$38 Million
Financing to support advancement of novel disease-modifying therapy targeting genetic diseases, including cystic fibrosis SAN DIEGO and REHOVOT, Israel, Aug. 2, 2017 -- (Healthcare Sales & Marketing Network) -- Sevion Therapeutics, Inc. (OTCQB: SVON) a... Biopharmaceuticals, Venture Capital Eloxx Pharmaceuticals, Cystic Fibrosis, Duchene Muscular Dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - August 2, 2017 Category: Pharmaceuticals Source Type: news

Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy
(AFM-T é l é thon) Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, after injecting microdystrophin (a 'shortened' version of the dystrophin gene) via a drug vector, the researchers managed to restore muscle strength and stabilise the clinical symptoms in dogs naturally affected by Duchenne muscular dystrophy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 25, 2017 Category: International Medicine & Public Health Source Type: news

Sarepta hikes full-year sales estimate for Duchenne drug
Sarepta Therapeutics reported Wednesday that sales of its Duchenne muscular dystrophy drug have picked up momentum since the FDA's controversial approval of the treatment last year. Cambridge-based Sarepta (Nasdaq: SRPT) said that the drug, Exondys 51, had generated $35 million in sales in the second quarter of 2017. That easily exceeded the consensus estimate of analysts, which was around $22 million. Sarepta also raised its guidance for total revenue in 2017, from more tha n $95 million to between… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 19, 2017 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

Sarepta hikes full-year sales estimate for Duchenne drug
Sarepta Therapeutics reported Wednesday that sales of its Duchenne muscular dystrophy drug have picked up momentum since the FDA's controversial approval of the treatment last year. Cambridge-based Sarepta (Nasdaq: SRPT) said that the drug, Exondys 51, had generated $35 million in sales in the second quarter of 2017. That easily exceeded the consensus estimate of analysts, which was around $22 million. Sarepta also raised its guidance for total revenue in 2017, from more tha n $95 million to between… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 19, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news

​BioMarin snags cash as Sarepta skips Duchenne patent battle
Sarepta Therapeutics Inc. agreed to pay $35 million plus royalties and potential milestones to BioMarin Pharmaceutical Inc. to resolve a dispute over patents on its Duchenne muscular dystrophy drug. Sarepta (NASDAQ: SRPT) said Tuesday it had reached an agreement to license the intellectual property of San Rafael-based BioMarin (NASDAQ: BMRN), which had challenged key patents covering Sarepta’s approved Duchenne drug, Exondys 51 as well as other so-called “exon-skipping” treatments. Tre nding:… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - July 18, 2017 Category: Health Management Authors: Max Stendahl Source Type: news

Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy
Designation Covers Broad Treatment of Duchenne Muscular Dystrophy (DMD) LOS ANGELES, July 18, 2017 -- (Healthcare Sales & Marketing Network) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing biological therapies for Duch... Biopharmaceuticals, FDA Capricor Therapeutics, Duchenne muscular dystrophy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 18, 2017 Category: Pharmaceuticals Source Type: news

​Sarepta skips over a Duchenne patent battle with $35M settlement
Cambridge-based Sarepta Therapeutics has agreed to pay $35 million plus royalties and potential milestones to a California biotech to resolve a dispute over patents on its Duchenne muscular dystrophy drug. Sarepta (Nasdaq: SRPT) said Tuesday that it had reached an agreement to license the intellectual property of San Rafael-based BioMarin Pharmaceutical (Nasdaq: BMRN). BioMarin had previously challenged key patents covering Sarepta’s approved Duchenne drug, Exondys 51, as well as other so-called… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 18, 2017 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

Mallinckrodt wins orphan drug status for long-acting muscular dystrophy therapy
Mallinckrodt Pharmaceuticals (NYSE:MNK) said today that the FDA granted orphan drug status to its long-acting cosyntropin acetate formulation, MNK-1411, for the treatment of Duchenne muscular dystrophy. In August last year, the FDA approved the company’s request to fast track its investigational new drug application for the therapy. Get the full story at our sister site, Drug Delivery Business News. The post Mallinckrodt wins orphan drug status for long-acting muscular dystrophy therapy appeared first on MassDevice. (Source: Mass Device)
Source: Mass Device - July 13, 2017 Category: Medical Devices Authors: Sarah Faulkner Tags: Food & Drug Administration (FDA) Pharmaceuticals Wall Street Beat Mallinckrodt Source Type: news

Tiny cellular antennae key to fat formation in muscle
(University of California - San Francisco) Like it or not, as we age, our muscle cells are slowly exchanged, one by one, for fat cells. This process quickens when we injure a muscle, and an extreme form of this process is also seen in muscle-wasting diseases such as Duchenne muscular dystrophy (DMD). Now, scientists at UC San Francisco have shown that cellular antennae called cilia, found on fat-forming cells interspersed in muscle, play a key role in this muscle-to-fat transformation. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - July 13, 2017 Category: International Medicine & Public Health Source Type: news

How FDA plans to help consumers capitalize on advances in science
By: Scott Gottlieb, M.D. We’re at a point in science where new medical technologies hold out the promise of better treatments for a widening number of vexing conditions. Over the last few decades, science has enabled fundamental advances in our understanding of the genetic and protein bases of human disease. These developments are already being translated into new medicines. In more cases, these treatments target the underlying mechanisms that drive different diseases. These advances hold out the promise of arresting and even curing a growing number of diseases. To build upon such opportunities, FDA will soon unveil ...
Source: Mass Device - July 11, 2017 Category: Medical Devices Authors: Danielle Kirsh Tags: Blog FDA Voice Source Type: news

Sarepta names former Allergan executive as new CEO
Sarepta Therapeutics, the Cambridge biotech that won approval last year for the first-ever Duchenne muscular dystrophy drug, said Wednesday that it had appointed former Allergan executive Douglas Ingram as its new CEO. Sarepta (Nasdaq: SRPT) had been searching for a new CEO after Ed Kaye announced in April that he planned to step down. Ingram served as president of Allergan until the company was acquired by Actavis in 2015. More recently, he was CEO of Chase Pharmaceuticals, a California biotech… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - June 28, 2017 Category: Health Management Authors: Max Stendahl Source Type: news

Sarepta names former Allergan executive as new CEO
Sarepta Therapeutics, the Cambridge biotech that won approval last year for the first-ever Duchenne muscular dystrophy drug, said Wednesday that it had appointed former Allergan executive Douglas Ingram as its new CEO. Sarepta (Nasdaq: SRPT) had been searching for a new CEO after Ed Kaye announced in April that he planned to step down. Ingram served as president of Allergan until the company was acquired by Actavis in 2015. More recently, he was CEO of Chase Pharmaceuticals, a California biotech… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 28, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news

Insurers Battle Families Over Costly Drug for Fatal Disease
The case of Exondys 51 poses emotionally charged issues for families of young boys with a rare illness, who are fighting companies to get coverage for an expensive drug approved on a lower bar of proof. (Source: NYT Health)
Source: NYT Health - June 22, 2017 Category: Consumer Health News Authors: KATIE THOMAS Tags: Drugs (Pharmaceuticals) Health Insurance and Managed Care Muscular Dystrophy Aetna Inc Food and Drug Administration Sarepta Therapeutics Inc. Prices (Fares, Fees and Rates) Source Type: news

14 Years Old. Losing Muscle Every Day.
Nicolas is 14 and has Duchenne muscular dystrophy, which is robbing him of his muscles — and his life. A new and expensive drug may help, but can he get it? (Source: NYT Health)
Source: NYT Health - June 22, 2017 Category: Consumer Health News Tags: Muscular Dystrophy Food and Drug Administration Drugs (Pharmaceuticals) Source Type: news