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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

Aquatic therapy for children with Duchenne muscular dystrophy: a pilot feasibility randomised controlled trial and mixed-methods process evaluation
Duchenne muscular dystrophy (DMD) is a rare disease that causes the progressive loss of motor abilities such as walking. Standard treatment includes physiotherapy. No trial has evaluated whether or not adding aquatic therapy (AT) to land-based therapy (LBT) exercises helps to keep muscles strong and children independent. To assess the feasibility of recruiting boys with DMD to a randomised trial evaluating AT (primary objective) and to collect data from them; to assess how, and how well, the intervention and trial procedures work. (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - June 21, 2017 Category: Consumer Health News Source Type: news

Sarepta Therapeutics and Genethon announce gene therapy research collaboration for Duchenne muscula
(AFM-T é l é thon) Sarepta Therapeutics, Inc. (NASDAQ:SRPT), and Genethon have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD). (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - June 21, 2017 Category: Biology Source Type: news

Muscle growth finding may assist with cancer treatment
(Monash University) Monash University's Biomedicine Discovery Institute (BDI) researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting in diseases including muscular dystrophy and cancer. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - June 14, 2017 Category: Cancer & Oncology Source Type: news

Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy
Duchenne muscular dystrophy is caused by a faulty gene that leads to progressive muscle weakness. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - June 13, 2017 Category: American Health Source Type: news

C-Path and CDISC announce public review period for Duchenne Therapeutic Area Standard
(Critical Path Institute (C-Path)) The Clinical Data Interchange Standards Consortium and Critical Path Institute announce the availability of a Duchenne muscular dystrophy therapeutic area user guide (TAUG-DMD v1.0) for public review. The review period for the TAUG-DMD v1.0 began on May 8, 2017, and runs through July 6, 2017. Qualified researchers and clinicians are encouraged to review TAUG-DMD v1.0 and offer feedback. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 13, 2017 Category: International Medicine & Public Health Source Type: news

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A new interactive tool is available to help parents assess whether their child ’s physical development is on target for their age.  Developed by the American Academy of Pediatrics (AAP), the Center for Disease Control and Prevention (CDC) and Parent Project Muscular Dystrophy, this tool is designed for parents who have concerns about their child ’s physical activity up to age  five.  Called the “Physical Developmental Delays: What to Look For” can be found athttp://motordelay.aap.org  and is provided at no cost.Sometimes it ’s very hard to figure out if there are delay...
Source: Pediatric Health Associates - June 9, 2017 Category: Pediatrics Tags: Healthy Habits Source Type: news

Moxley, a Pioneer in Muscular Dystrophy Research and Care, to Step Down
Richard (Dick) Moxley III, M.D., one of the founding fathers of the University of Rochester Medical Center Department of Neurology, is retiring after a career spanning more than four decades. Moxley, who holds the Helen Aresty Fine and Irving Fine Professorship in Neurology, is widely recognized for helping revolutionize our understanding of muscular dystrophy and the care of patients with this disease. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - June 8, 2017 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news

Webinar Recording: Nationwide Children ’s Hospital Carrier Study
PPMD recently hosted a webinar for a discussion on the Nationwide Children ’s Hospital Carrier Study. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 30, 2017 Category: Neurology Source Type: news

Reporter sues FDA for documents related to Sarepta drug approval
A journalist has filed a federal lawsuit against the FDA seeking documents related to the agency ’s controversial approval last year of Exondys 51, a Duchenne muscular dystrophy treatment developed by Cambridge-based Sarepta Therapeutics. Charles Seife, a journalist and professor at New York University, filed a complaint Thursday in Manhattan federal court challenging the FDA’s decision no t to disclose records that he requested under the Freedom of Information Act. The documents relate to the… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 25, 2017 Category: American Health Authors: Max Stendahl Source Type: news

Reporter sues FDA for documents related to Sarepta drug approval
A journalist has filed a federal lawsuit against the FDA seeking documents related to the agency ’s controversial approval last year of Exondys 51, a Duchenne muscular dystrophy treatment developed by Cambridge-based Sarepta Therapeutics. Charles Seife, a journalist and professor at New York University, filed a complaint Thursday in Manhattan federal court challenging the FDA’s decision no t to disclose records that he requested under the Freedom of Information Act. The documents relate to the… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 25, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news

​Bain Capital rakes in $720M for first life sciences fund
Bain Capital said Tuesday it has raised $720 million for its first fund devoted solely to life science companies, a project led by former Biogen executive Adam Koppel. The fund, called Bain Capital Life Sciences, has already made its presence felt in the local biotech cluster. On March 30, the fund, along with RA Capital Management, led a $50 million Series C round for Solid Biosciences, which is working on gene therapy treatments for Duchenne muscular dystrophy . That same day, Bain’s life science… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 23, 2017 Category: American Health Authors: Max Stendahl Source Type: news

Bain Capital raises $720m life sciences fund
Bain Capital said today that it raised $720 million for its 1st investment fund focused exclusively on the life sciences sector. “We are excited by some of the long-term secular trends in the life sciences space,” Bain Capital Life Sciences managing director Adam Koppel said. “Big pharma is increasingly outsourcing R&D at the same time as many new technologies and treatments are being developed.” The Boston-based fund has already backed a pair of companies, RNAi inhibitor maker Dicerna Pharmaceuticals (NSDQ:DRNA) and Solid Biosciences, which is developing treatments for Duche...
Source: Mass Device - May 23, 2017 Category: Medical Devices Authors: Brad Perriello Tags: Funding Roundup Wall Street Beat Bain Capital Venture Capital/Private Equity Source Type: news

FDA Pediatric Advisory Committee Unanimously Votes YES — Now Moves to Commissioner
Today ’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE clinical trial. A final decision will be announced once the Commissioner has reviewed the summary report. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 18, 2017 Category: Neurology Source Type: news

Weekly steroids strengthen and repair muscles
(Northwestern University) In a surprising finding, weekly doses of glucocorticoid steroids, such as prednisone, help speed recovery in muscle injuries, reports a new study. The weekly steroids also repaired muscles damaged by muscular dystrophy. When given daily over long periods, prednisone can cause muscle wasting. But the once weekly doses of the steroid increased proteins that stimulate muscle repair. The studies were conducted in mice, with broad implications for humans. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 16, 2017 Category: International Medicine & Public Health Source Type: news

Deciphering What We Know About EMFLAZA
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 15, 2017 Category: Neurology Source Type: news

ACTION ALERT: Urge Your Senators to Sign the FY18 Duchenne Funding Letter
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 15, 2017 Category: Neurology Source Type: news

Capricor Therapeutics Provides Update on ALLSTAR Trial
Unlikely to Achieve Primary Efficacy Endpoint of Change in Infarct Size in Patients Following Heart Attack Capricor to Focus on Potential Registration Program in Duchenne Muscular Dystrophy Company to Hold Conference Call Today at 5:00 AM PDT / 8:00 ... Regenerative Medicine, Cardiology Capricor Therapeutics, ALLSTAR Trial, allogeneic cardiosphere-derived cells (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - May 12, 2017 Category: Pharmaceuticals Source Type: news

In Honor of Moms
This Mother ’s Day, thank someone for their role in the fight to end Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 10, 2017 Category: Neurology Source Type: news

Webinar Recording: Results from Phase I/II HOPE Clinical Trial of CAP-1002
PPMD, Coalition Duchenne, and Capricor recently hosted a webinar to hear a community update on the six-month results from Capricor's randomized Phase I/II HOPE Clinical Trial of CAP-1002. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 9, 2017 Category: Neurology Source Type: news

PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access
Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. PPMD will continue to monitor and remain engaged in the access environment in order to understand the impact of every new approval, including EMFLAZA, and will update our online Access Resource as information becomes available. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 8, 2017 Category: Neurology Source Type: news

Meet the Race to End Duchenne .1K!
September is Duchenne Action Month, and now is the time for you to start planning an event than can raise awareness and money, while providing people with an amazing experience that is fun and rewarding …the Race to End Duchenne .1K! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 5, 2017 Category: Neurology Source Type: news

Webinar Recording: Givinostat in Duchenne
PPMD and Italfarmaco recently hosted a webinar to discuss Italfarmaco's investigational drug Givinostat and how it works in Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 1, 2017 Category: Neurology Source Type: news