http://www.mda.org/whatsnew/ 12/23/2009 Get the latest research news from the Muscular Dystrophy Association (MDA) en-us http://quest.mda.org/news/new-grant-lgmd2d-gene-therapy 12/23/2009 Early results show that when AVI4658 is delivered system-wide through the bloodstreams of boys with DMD, it’s safe and increases dystrophin production. http://quest.mda.org/news/exon-skipping-drug-delivers-again 12/23/2009 Early results show that when AVI4658 is delivered system-wide through the bloodstreams of boys with DMD, it’s safe and increases dystrophin production. http://quest.mda.org/news/new-mda-grant-will-help-develop-fa-drug 12/17/2009 Repligen Corp. is proceeding with development of an experimental compound to treat Friedreich's ataxia, with help from a new MDA grant. http://quest.mda.org/news/big-horses-big-time 12/17/2009 Thanks to his love of horse-pulling competitions, a Kentucky youth with DMD recently was named Tri-State Horse Person of the Year. http://quest.mda.org/news/internship-opportunities-college-students 12/17/2009 Dec. 31, 2009, is the deadline to apply for the 2010 Emerging Leaders Summer Internship Program for College Students with Disabilities. http://quest.mda.org/news/als-research-restoring-disrupted-connections 12/17/2009 A chemical called microRNA 206 appears to help re-establish nerve-muscle connections after injury in ALS research mice. http://quest.mda.org/news/two-als-drugs-show-early-promise 12/10/2009 The experimental drugs KNS760704 and SB509 both pass safety tests and seem to improve function for ALS patients. http://quest.mda.org/news/international-als-conference 12/09/2009 Daily Web reports are available from a multinational ALS symposium held in Berlin Dec. 8-10, 2009. http://quest.mda.org/news/als-research-poison-dirt 12/09/2009 "Toxic desert dust" may be why military personnel serving in the 1990-1991 Gulf War experienced abnormally high rates of ALS, new findings suggest. http://quest.mda.org/news/als-drug-trial-restarted 12/09/2009 After a two-year review, the FDA approves reopening a clinical trial of the experimental compound arimoclomol. http://quest.mda.org/news/new-dmd-care-recommendations 12/03/2009 After a year of planning, new recommendations for physicians caring for DMD patients have been released. http://www.mda.org/publications/Quest/extra/nov09/lgmd-tgf.html 11/19/2009 A change in the LTBP4 protein gene reduces muscle-damaging signals in mice with a disease resembling LGMD2C. http://www.mda.org/publications/Quest/extra/nov09/lgmd-tgf.html 11/19/2009 A change in the LTBP4 protein gene reduces muscle-damaging signals in mice with a disease resembling LGMD2C. http://www.mda.org/publications/Quest/extra/nov09/sma-drug-candidate.html 11/19/2009 Researchers have identified a compound that helps cells produce more full-length SMN protein from the backup SMN2 gene. http://www.mda.org/publications/Quest/extra/nov09/stop-smoking.html 11/19/2009 A new review of the medical literature confirms that smoking significantly raises the risk of developing ALS. http://www.mda.org/publications/Quest/extra/nov09/follistatin.html 11/12/2009 Injections of genes for the muscle-growth protein follistatin strengthened leg muscles in monkeys. http://www.mda.org/research/091112gene-therapy-primates.html 11/11/2009 Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced today. http://www.mda.org/publications/Quest/extra/nov09/als-lithium.html 11/9/2009 An Italian ALS lithium trial is ended after an interim analysis revealed no benefit and possible toxicity. http://www.mda.org/publications/Quest/extra/nov09/compound_mmd.html 11/4/2009 A small-molecule compound counteracts some of the effects of abnormal genetic instructions in MMD1. http://www.mda.org/publications/Quest/extra/Oct09/ALSThinkTank.html 10/28/2009 Nine leaders in the field of ALS research put their heads together on Oct. 24, 2009 http://www.mda.org/publications/Quest/extra/Oct09/ALS-APC.html 10/27/2009 A compound (APC) used to treat blood infections in humans seems to improve ALS symptoms in mice. http://www.mda.org/publications/Quest/extra/Oct09/avi-biopharma-trial.html 10/27/2009 DMD participants are sought for the first U.S. exon skipping trial, to begin in March 2010. http://www.mda.org/research/091022new-dmd-treatment.html 10/22/2009 A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). http://www.mda.org/publications/Quest/extra/Oct09/exon-skipping-dmd.html 10/21/2009 Exon skipping proves effective in mice with a severe DMD-like disease. http://www.mda.org/publications/Quest/extra/Oct09/ALS-ISIS.html 10/16/2009 The first trial to block SOD1 protein in ALS using "antisense" will open by the end of 2009. http://www.mda.org/publications/Quest/extra/Oct09/als-tdi-webcast.html 10/16/2009 An ALS compound that's ready for biotech development is described in this Oct. 5, 2009, Webcast. http://www.mda.org/publications/Quest/extra/Oct09/exon-skipping.html 10/16/2009 A multinational pharmaceutical company has committed to developing Prosensa's exon skipping compound for DMD. http://www.mda.org/research/091012NIH-ARRA.html 10/12/2009 At least 15 Muscular Dystrophy Association research grantees, past and present, have won funding from the National Institutes of Health through the American Recovery and Revitalization Act (ARRA) to accelerate the search for treatments and cures for neuromuscular diseases. http://www.als-mda.org/research/news/091013pt-guidelines.html 10/12/2009 New guidelines for the most effective treatments for people with amyotrophic lateral sclerosis (ALS) will be released by the American Academy of Neurology (AAN) tomorrow. http://www.mda.org/publications/Quest/extra/Oct09/ALS-TDP43.html 10/12/2009 A new type of ALS research mouse, with a mutation in the TDP43 gene, has been developed. http://www.mda.org/publications/Quest/extra/Oct09/ALSRegistryFAQ.html 10/8/2009 Questions and answers about ALS and the upcoming National ALS Registry have been posted online. http://www.mda.org/publications/Quest/extra/Oct09/athena-dna-tests.html 10/7/2009 Athena Diagnostics can now analyze five genes for mutations that cause familial ALS. http://www.mda.org/publications/Quest/extra/Oct09/dmd-idebenone.html 10/7/2009 Idebenone, an antioxidant and possible cellular energy booster, will be tested in some 240 boys with DMD. http://www.mda.org/publications/Quest/extra/sept09/target.html 9/29/2009 A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake. http://www.mda.org/publications/Quest/extra/sept09/enhancing.html 9/25/2009 Eliminating a protein enhanced cellular cleanup, saved nerve cells and prolonged survival in female ALS mice. http://www.mda.org/publications/Quest/extra/sept09/mutation.html 9/25/2009 A variant in the SMN2 gene leads to more full-length SMN protein and better function. http://www.mda.org/publications/Quest/extra/sept09/als-lithium.html 9/24/2009 Although the National Institutes of Health cut short its lithium trial, MDA’s will continue for now. http://www.als-mda.org/research/news/090923als-neuralstem.html 9/23/2009 The first FDA-approved human stem cell trial to treat amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease, will be conducted at the Muscular Dystrophy Association ALS Center at Emory University in Atlanta. http://www.mda.org/publications/Quest/extra/sept09/als-risk.html 9/18/2009 A multinational team found three DNA variations that may increase the risk of developing ALS. http://www.mda.org/publications/Quest/extra/sept09/obesity.html 9/18/2009 GW501516, being developed to treat obesity, increases levels of the useful muscle protein utrophin in mice. http://www.mda.org/publications/Quest/extra/sept09/dmd-delays.html 9/4/2009 A new survey shows an average of 2.5 years elapse between symptom onset and diagnosis of DMD. http://www.mda.org/publications/Quest/extra/aug09/prevention.html 8/26/2009 A new assisted-reproduction strategy may prevent transmission of mitochondrial disease. http://www.mda.org/publications/Quest/extra/aug09/tau.html 8/26/2009 New evidence casts doubt on the role of tau protein in inclusion-body myositis. http://www.mda.org/publications/Quest/extra/aug09/abnormal.html 8/26/2009 New findings show abnormally activated parts of a gene called DUX4 may underlie FSH dystrophy. http://www.mda.org/publications/Quest/extra/aug09/place.html 8/21/2009 High levels of misplaced proteins may worsen the disease process in MG. http://www.mda.org/publications/Quest/extra/aug09/path.html 8/18/2009 FDA launches ‘Access to Investigational Drugs’ Web site to aid patients and doctors. http://www.mda.org/publications/Quest/extra/aug09/sbma.html 8/14/2009 Mice with an SBMA-like disease benefited from extra IGF1 genes in their muscle fibers. http://www.mda.org/publications/Quest/extra/aug09/cmd.html 8/14/2009 An Aug 15-16 conference in Philadelphia for families affected by CMD can be watched online. http://www.mda.org/publications/Quest/extra/aug09/outbursts.html 8/14/2009 World Federation of Neurology teams up with Twitter to untangle unconventional ALS treatments. http://www.mdavp.org/newsletter/MVP_Quarterly_Summer.pdf 8/10/2009 MDA Venture Philanthropy has published its Quarterly Summer Newsletter. http://www.mda.org/publications/Quest/extra/jul09/iplex.html 7/28/2009 Insmed halts development of its drug Iplex for MMD and ALS, at least for now. http://www.mda.org/publications/Quest/extra/jul09/pipeline.html 7/28/2009 An ALS TDI Webcast summarizes research progress as of July 2009. http://www.mda.org/publications/Quest/extra/jul09/cmt1a.html 7/28/2009 The jury is still out on vitamin C for treating CMT1A symptoms http://www.mda.org/publications/Quest/extra/jul09/bmd.html 7/17/2009 Physicians, scientists and advocates to discuss BMD in Boston conference Aug. 1. http://www.mda.org/publications/Quest/extra/jul09/restored.html 7/17/2009 MDA advocates help convince legislators to ease restrictions in proposed bill. http://www.mda.org/research/090716synthetic-molecule.html 7/16/2009 Researchers have found a critical key to blocking the cause of myotonic muscular dystrophy by fooling defective genetic material in a discovery that could have major implications for finding a treatment for the disease, the Muscular Dystrophy Association announced today. http://www.mda.org/research/090710MDA-grants.html 7/13/2009 At a time when federal and private funds for biomedical research have become scarce, the Muscular Dystrophy Association reasserts its leadership in the fight against muscle diseases by announcing grants to innovative research projects throughout America and in Canada. http://www.mda.org/publications/Quest/extra/jul09/pon.html 7/10/2009 ALS Research: No Single PON Gene Variant Raises Risk of ALS http://www.mda.org/publications/Quest/extra/jul09/mtm-cnm.html 7/10/2009 Families affected by centronuclear myopathies will Webcast their conference in Houston July 24-26. http://www.mda.org/publications/Quest/extra/jun09/mmd1.html 6/26/2009 The drug Iplex, developed by the Richmond, Va., biopharmaceutical company Insmed, did not improve muscle function, strength or endurance in a phase 2 trial in type 1 myotonic dystrophy (MMD1, or DM1), the company announced June 25, 2009. http://www.mda.org/research/090625insmed-iplex-results.html 6/25/2009 Insmed Inc. (NASDAQ CM: INSM), a biopharmaceutical company, today announced results from its exploratory U.S. Phase II clinical trial evaluating IPLEX™ (mecasermin rinfabate) in patients with myotonic muscular dystrophy ("MMD"). http://www.mda.org/publications/Quest/extra/jun09/pond_scum.html 6/22/2009 Recent media reports have raised the question of a possible link between an increased risk of developing amyotrophic lateral sclerosis (ALS) and living near Lake Mascoma in Western New Hampshire. http://www.mda.org/publications/Quest/extra/jun09/protein_repair.html 6/19/2009 Scientists in the United States and Japan have identified a three-protein cluster that reseals damaged muscle-fiber membranes. http://www.mda.org/publications/Quest/extra/jun09/cardiac.html 6/12/2009 A protein present in skeletal muscles during fetal development and in the heart after birth can apparently compensate for a similar protein that's missing in a small percentage of patients with the muscle disease known as nemaline myopathy. http://www.mda.org/publications/Quest/extra/jun09/WNT7a.html 6/12/2009 In experiments in mice, Michael Rudnicki, an MDA grantee at the Sprott Center for Stem Cell Research at Ottawa Hospital Research Institute (OHRI), and colleagues, found the WNT7a protein stimulates muscle repair by causing proliferation (an increase in number) of "satellite stem cells." http://www.mda.org/research/090611stem-cell-research.html 6/11/2009 A discovery that strengthens the body's ability to repair muscle tissue could lead to new treatments for people with muscular dystrophy and other degenerative muscle diseases. http://www.mda.org/publications/Quest/extra/jun09/respiratory.html 6/3/2009 The proceedings of a symposium titled "Pulmonary Management of Pediatric Patients with Neuromuscular Disorders" have been published as a supplement to the May 2009 issue of the journal Pediatrics. http://www.mda.org/research/090429griggs-aan-president.html 4/29/2009 Neurologist Robert Griggs, a longtime Muscular Dystrophy Association (MDA) research grantee and MDA-affiliated clinician at the University of Rochester (N.Y.) Medical Center, has been elected 31st president of the American Academy of Neurology (AAN). http://www.mda.org/research/090413lgmd-gene-therapy.html 4/15/2009 Researchers supported by the Muscular Dystrophy Association (MDA) and the National Institutes of Health say results of a gene therapy trial in three people with limb-girdle muscular dystrophy (LGMD) are the first to show promise beyond safety alone, MDA announced today. http://www.mda.org/research/090316dmd-exon-skipping.html 3/16/2009 Scientists have successfully treated dogs with a disease closely resembling Duchenne muscular dystrophy (DMD) , using a molecular treatment strategy called "exon skipping," the Muscular Dystrophy Association (MDA) announced today. The strategy is simultaneously under development in human patients. http://www.als-mda.org/research/news/090310als-iplex-trial.html 3/10/2009 The Food and Drug Administration (FDA) announced today that it would work closely with the biopharmaceutical company Insmed of Richmond, Va., to develop a clinical trial of Iplex, an experimental medication developed by the company, in patients with ALS (amyotrophic lateral sclerosis). http://www.mda.org/research/090306dmd-stem-cells.html 3/05/2009 A new type of stem cell that may have implications for treating several muscle diseases has been identified by scientists at the University of Colorado at Boulder and at the University of Colorado Health Sciences Center in Aurora, the Muscular Dystrophy Association announced today. http://www.als-mda.org/research/news/090227als-fus-gene.html 2/27/2009 Two independent research teams, one based in the United States and Canada and the other in the United Kingdom and Australia, have identified mutations in a gene called FUS on chromosome 16 as a cause of familial amyotrophic lateral sclerosis (ALS). http://www.als-mda.org/research/news/090203lou-gehrig-anniv.html 2/03/2009 Major League Baseball will raise awareness and financial support for organizations leading the fight against ALS (Amyotrophic Lateral Sclerosis), otherwise known as "Lou Gehrig's Disease" with a new charitable campaign, "4 ♦ ALS Awareness," Baseball Commissioner Allan H. (Bud) Selig announced today. http://www.mda.org/research/090202edmd-nmj-involvement.html 2/02/2009 A multinational team coordinated by Tom Misteli at the National Cancer Institute of the National Institutes of Health in Bethesda, Md., has found that, in mice with a disease resembling Emery-Dreifuss muscular dystrophy (EDMD), lamin A or C protein defects interfere with the way cell nuclei normally localize in skeletal-muscle fibers at the point where each fiber receives signals from a nerve cell. http://www.mda.org/news/090129sma_patient_registry.html 1/29/2009 You can join the International Spinal Muscular Atrophy Patient Registry online! The Registry website allows individuals to participate in the Registry completely through on-line interaction including learning about and signing up for new research study opportunities. http://www.mda.org/research/090128cmd-treatment-ideas.html 1/28/2009 Recently published findings from two independent groups have suggested possible treatment development pathways for the merosin-deficient and integrin-deficient forms of congenital muscular dystrophy (CMD). http://www.mda.org/research/090123dmd-exon-skipping-trial.html 1/23/2009 On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom. http://www.als-mda.org/research/news/090120als-new-therapeutic-delivery.html 1/20/2009 ALS Therapy Development Institute (ALS TDI) and Asklepios BioPharmaceutical, Inc. announced today their collaboration aimed at developing a panel of viral vectors that could be used to deliver therapeutics based on targets identified by ALS TDI that slow or stop amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease). http://www.mda.org/research/090105ResearchersExplore.html 1/5/2009 Revving up a natural cellular defense mechanism that neurons (nerve cells) use to protect themselves looks like a good lead in developing treatments for spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) and perhaps similar neurodegenerative diseases, new research shows.