Claim: MUSCULAR DYSTROPHY: HEARTBROKEN FAMILY AWAITS FDA DECISION ON EXPERIMENTAL DRUG
Verdict: SUPPORTS

Retrieved passages:
title: Henri Termeer
text: Duchenne muscular dystrophy (DMD), is being developed by pharmaceutical giant, GlaxoSmithKline (GSK). GSK, which also makes major investments in rare diseases and orphan drugs, licensed 051 in 2009 for "$25 million upfront and another $655 million in milestones." "Prosensa has a technology that could provide an array of RNA therapies for different variants of DMD, which affects about 1 in 3,500 male births and causes muscle wasting that leads to premature death." In 2012 Prosensa was listed by the industry news monitor Fierce Biotech's Fierce 15 as one of their top choices of pharmaceutical firms. Fierce Biotech covers biotechnology news

title: Ezutromid
text: lack of dystrophin to retard muscle degeneration. Both the FDA and European Medicines Agency has given ezutromid an orphan drug designation. The FDA Office of Orphan Products and Development offers an Orphan Drug Designation program (ODD) that allows drugs aimed to treat diseases that affect less than 200,000 people in the U.S. monetary incentives such as a period of market exclusivity, tax incentives, and expedited approval processes. The first Phase 1b trial (NCT02056808) began on November 2013 and involved 12 patients aged 5–11 years old. The patients were divided into three groups given escalating oral doses testing the safety and

title: Becker's muscular dystrophy
text: is a known inhibitor of the protein cyclophilin D, which regulates the swelling of mitochondria in response to cellular injury. Researchers decided to test the drug in mice engineered to carry MD after earlier laboratory tests showed deleting a gene that encodes cycolphilin D reduced swelling and reversed or prevented the disease’s muscle-damaging characteristics. According to a review by Bushby, et al. if a primary protein is not functioning properly then maybe another protein could take its place by augmenting it. Upregulation of compensatory proteins has been done in models of transgenic mice. The progression of Becker muscular dystrophy is

title: Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013
text: to various forms of muscular dystrophy, including Duchenne, Becker, limb girdle, congenital, facioscapulohumeral, myotonic, oculopharyngeal, distal, and Emery–Dreifuss muscular dystrophies. According to the Muscular Dystrophy Association, "since 2001, there have been 67 clinical trials of drugs or therapies for muscular dystrophy, and currently 37 clinical trials are under way." "This summary is based largely on the summary provided by the Congressional Research Service, a public domain source." The Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013 would amend the Public Health Service Act to revise the muscular dystrophy research program of the National Institutes of

title: Gene therapy
text: levels of SERCA2, a protein in heart muscles, improving muscle function. The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process. In 2016 it was reported that no improvement was found from the CUPID 2 trial. In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 7–32 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills. The other children had Wiskott-Aldrich syndrome, which leaves them to open to

title: Drisapersen
text: review with the US Food and Drug Administration (FDA). In January 2016, the FDA rejected drisapersen (Kyndrisa) after it concluded that the standard of substantial evidence of effectiveness had not been met, effectively shifting focus to rival Sarepta's eteplirsen. The Prescription Drug User Fee Act (PDUFA) goal dates for these are December 27, 2015 for drisapersen and February 26, 2016 for eteplirsen. In May 2016, BioMarin announced they intended to discontinue clinical and regulatory development of drisapersen as well as related first generation compounds currently in Phase 2 studies for distinct forms of Duchenne muscular dystrophy. The company claim they

title: Muscular Dystrophy Association
text: some of the diseases in MDA's program, the availability of a newborn screening program at the time of treatment availability presents the best opportunity for impacting optimal and potential lifesaving treatment outcomes." The MDA supported the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013 (H.R. 594; 113th Congress), a bill that would amend the Public Health Service Act to revise the muscular dystrophy research program of the National Institutes of Health (NIH). MDA argued that "a great deal of work still needs to be done, and increased federal support is needed to ensure that researchers

title: Duchenne muscular dystrophy
text: disease. Current research includes exon-skipping, stem cell replacement therapy, analog up-regulation, gene replacement, and supportive care to slow disease progression. Efforts are ongoing to find medications the either return the ability to make dystrophin or utrophin. Other efforts include trying to block the entry of calcium ions into muscle cells. Antisense oligonucleotides (oligos), structural analogs of DNA, are the basis of a potential treatment for 10% of people with DMD. The compounds allow faulty parts of the dystrophin gene to be skipped when it is transcribed to RNA for protein production, permitting a still-truncated but more functional version of the

title: Muscular Dystrophy Association
text: store. MDA targets the following muscle-affecting diseaes: The organization also targets muscle diseases due to deficiencies in carnitine and the following enzymes: The MDA supported the Newborn Screening Saves Lives Reauthorization Act of 2013 (H.R. 1281; 113th Congress), a bill that would amend the Public Health Service Act to reauthorize grant programs and other initiatives to promote expanded screening of newborns and children for heritable disorders. The MDA argued that "many of the drug therapies currently under development for MDA's community will be of most benefit if administered either presymptomatically or early in the progression of the disease. Thus, for

title: Ataluren
text: Commission to treat people with nonsense mutation Duchenne muscular dystrophy in August 2014; a confirmatory phase III clinical trial was required. By December it was on the market in Germany, France, Italy, Denmark, Spain and a number of other European countries. In February 2016, FDA declined to accept PTC Therapeutics new drug application for ataluren, which was based on a clinical trial in which ataluren missed its primary endpoint; PTC appealed and the FDA declined again in October 2016. In July 2016, NHS England agreed a Managed Access Agreement (MAA) for Translarna providing reimbursed patient access to Translarna in England

title: Sarepta Therapeutics
text: polycystic kidney disease, redirection of drug metabolism, treatment of some mutations causing Duchenne muscular dystrophy (DMD), and inhibition of infectious diseases. Their greatest clinical & commercial success thus far has been in treatment of DMD. A new class of Morpholino oligos, the peptide-linked Morpholinos or PPMO, are linked to an arginine-rich cell-penetrating peptide to enhance their delivery into cells and have entered clinical trials The Morpholino drug eteplirsen, targeting exon 51 of the dystrophin mRNA, has been approved as a human therapeutic by the FDA in 2016 and clinical trials for Morpholinos targeting other exons are ongoing. Morpholinos have been

title: Marathon Pharmaceuticals
text: patients usually die by age 25, although recently some people have lived into their 30s and 40s with assistive devices and support. On February 13, 2017, the CEO of Marathon, Jeff Aronin, announced that after four years of development and over 17 trials, the company was granted FDA approval for deflazacort (Emflaza) to treat Duchenne muscular dystrophy, a rare disease that affects approximately 15,000 young boys in the U.S. Prior to the FDA approval, less than 10% of Duchenne patients in the U.S. had access to Emflaza. Post FDA approval, all Duchenne patients have access to an FDA approved and

title: Muscular Dystrophy Community Assistance Research and Education Amendments of 2001
text: pass the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013 (H.R. 594; 113th Congress), a bill that would amend the Public Health Service Act to revise the muscular dystrophy research program of the National Institutes of Health (NIH). The bill would amend the Muscular Dystrophy Community Assistance Research and Education Amendments of 2001 to authorize the Secretary of Health and Human Services to: (1) update and disseminate widely existing Duchenne-Becker muscular dystrophy care considerations for pediatric patients, and (2) develop and disseminate widely Duchenne-Becker muscular dystrophy considerations for adult patients and acute care considerations for

title: Duchenne muscular dystrophy
text: to develop therapies for DMD in the future. In 2007, researchers did the world's first clinical (viral-mediated) gene therapy trial for Duchenne MD. Biostrophin is a delivery vector for gene therapy in the treatment of Duchenne muscular dystrophy and Becker muscular dystrophy. Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy. The symptom of muscle weakness usually begins around the age of four in boys and worsens quickly. Typically muscle loss occurs first in the upper legs and pelvis followed by those of the upper arms. This can result in trouble standing up. Most are

title: Ataluren
text: the biological target of ataluren is not known. Phase I clinical trials started in 2004. In 2010, PTC Therapeutics released preliminary results of its phase 2b clinical trial for Duchenne muscular dystrophy, with participants not showing a significant improvement in the six minute walk distance after the 48 weeks of the trial. This failure resulted in the termination of a $100 million deal with Genzyme to pursue the drug. In May 2014 ataluren received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and received market authorization from the European

title: Antisense therapy
text: received regulatory approval from FDA for use to treat spinal muscular atrophy. In September 2016 eteplirsen, a Morpholino oligo, received FDA approval for the treatment of Duchenne muscular dystrophy. Volanesorsen is in phase 3 clinical trials for treating hypertriglyceridemia as of December 2016. Because nucleases that cleave the phosphodiester linkage in DNA are expressed in almost every cell, unmodified DNA molecules are generally degraded before they reach their targets. Therefore, antisense drug candidate molecules are generally modified during the drug discovery phase of their development. Additionally, most targets of antisense are located inside cells, and getting nucleic acids across cell

title: Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013
text: a voice vote. The United States Senate voted on September 26, 2014 to pass the bill with unanimous consent. On September 26, 2014, President Barack Obama signed the bill into law and it became . The Parent Project Muscular Dystrophy supported the bill, urging supporters to take action to help them pass the bill. According to the organization, the bill would "update the landmark MD-CARE Act law that has done so much over the past 13 years to extend and improve life for those impacted by Duchenne and other forms of Muscular Dystrophy and to spur breakthroughs in research that

title: Omigapil
text: not yet available, omigapil is ready for human clinical trials to help mediate CMD. Omigapil has undergone extensive clinical trial scrutiny for Parkinson's disease and ALS, which indicates that the drug is safe to begin clinical trials for congenital muscular dystrophy. It has been investigated in the context of ketamine-like rapid acting antidepressant. Omigapil Omigapil (TCH346 or CGP3466) is a drug that was developed by Novartis and tested in clinical trials for its ability to help treat Parkinson's disease (PD) and amyotrophic lateral sclerosis (ALS). The development for PD and ALS have been terminated due to lack of benefit, but

title: Drisapersen
text: will continue to look into DMD and the next generation oligonucleotides. Drisapersen Drisapersen (also known as Kyndrisa, PRO051 and GSK2402968) is an experimental drug that was under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. The drug is a 2'-O-methyl phosphorothioate oligonucleotide that alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA. Duchenne muscular dystrophy (DMD) is caused when a mutation in the dystrophin gene changes the RNA so that it no longer codes for functional dystrophin protein. This usually happens due to a mutation that alters

title: Ataluren
text: via a five-year MAA. This followed a positive recommendation from the National Institute for Health and Care Excellence (NICE) in April 2016, subject to PTC and NHS England finalizing the terms of the MAA. NICE issued its final guidance later in July with implementation of the MAA for patients following within two months. In March 2017 PTC terminated development of ataluren for cystic fibrosis due to lack of efficacy in the Phase III trials. Ataluren Ataluren, formerly known as PTC124, is a pharmaceutical drug for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics and is sold