http://www.mda.org/whatsnew/ 8/30/2010 Get the latest research news from the Muscular Dystrophy Association (MDA) en-us http://quest.mda.org/news/decoy-receptor-lures-myostatin-helps-muscles 9/7/2010 Inserting a gene for a decoy myostatin receptor led to increased muscle size and strength in mice with a DMD-like disease http://quest.mda.org/news/leuprorelin-fails-improve-swallowing-sbma 8/30/2010 A trial of a drug that reduces testosterone levels did not improve swallowing in men with spinal-bulbar muscular atrophy. http://quest.mda.org/news/nationwide-childrens-hospital-named-md-research-center 8/27/2010 A $7 million federal grant will allow researchers to focus on solving immune-system obstacles to MD gene therapy. http://alsn.mda.org/news/als-research-briefs 8/27/2010 Short updates on ALS research: KNS-760704, FUS protein in stress granules, SOD1 and mitochondria. http://quest.mda.org/news/study-seeks-people-uncertain-md-diagnoses 8/27/2010 Researchers seek adults with nonspecific diagnoses of muscular dystrophy or myopathy, who might have late-onset Pompe disease. http://quest.mda.org/news/research-briefs-als-cmd-fa-and-sma 8/27/2010 News on amyotrophic lateral sclerosis, congenital muscular dystrophy, Friedreich's ataxia and spinal muscular atrophy. http://alsn.mda.org/news/expanded-ataxin-2-genes-major-contributor-als-risk 8/24/2010 Small expansions in the ataxin 2 gene significantly increase the risk of developing of ALS. http://quest.mda.org/news/research-briefs-als-bmd-dmd-mmd-sma-muscle-regeneration 8/20/2010 News on amyotrophic lateral sclerosis; Duchenne, Becker and myotonic muscular dystrophies; spinal muscular atrophy and muscle regeneration. http://quest.mda.org/news/not-one-two-dna-changes-are-needed-cause-fshd 8/19/2010 Researchers learn that two DNA changes on chromosome 4 — a contracted DNA segment and a "permissive" DNA signal — are needed to cause FSHD. http://www.mda.org/research/100819_fshd_dna_changes.html 8/19/2010 The Muscular Dystrophy Association today heralds a landmark muscular dystrophy advance by an international study team of scientists and physicians from the Netherlands, United States, France and Spain. http://alsn.mda.org/news/als-experts-urge-caution-regarding-head-injury-findings 8/18/2010 A small study has proposed that head injuries can cause an ALS-like disease, but MDA advisers say the evidence is insufficient. http://quest.mda.org/news/mda-awards-more-14-million-research-grants 8/18/2010 Thirty-eight new grants support research in ALS, central core disease, spinal muscular atrophy, muscular dystrophy, and other diseases in MDA's program http://www.mda.org/research/100818_mda-research-grants.html 8/18/2010 The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants. http://alsn.mda.org/news/more-fus-related-als-cases-found 8/13/2010 Investigators have identified many more cases of ALS due to mutations in the gene for FUS in a variety of ethnic groups. http://alsn.mda.org/news/blocking-protein-extends-survival-als-mice 8/12/2010 Mice with an SOD1 mutation and an ALS-like disease survived longer after researchers blocked the immune-system protein interleukin-1-beta. http://alsn.mda.org/news/learn-more-about-ceftriaxone-and-als 8/12/2010 Investigators on a study of ceftriaxone in amyotrophic lateral sclerosis (ALS) will host an informational webinar Tuesday, Sept. 7, 2010. http://quest.mda.org/news/research-briefs-als-dmd-mtm-and-sma 8/12/2010 Reports on research in amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), myotubular myopathy (MTM) and spinal muscular atrophy (SMA). http://alsn.mda.org/news/als-causing-tdp43-overstays-its-welcome 8/4/2010 Researchers have found that some ALS-related TDP43 mutations cause the TDP43 protein to hang around too long, which can be toxic to cells. http://alsn.mda.org/news/als-tdi-posts-quarterly-research-update 8/2/2010 New thinking about where ALS starts and how it develops was among the many topics discussed during ALS TDI’s webinar covering research in the second quarter of 2010. http://alsn.mda.org/news/study-probe-oxidative-stress-people-als 07/26/2010 Participants are being sought for a 12-center study that will follow people with ALS over time to examine relationships between the disease and oxidative stress. http://alsn.mda.org/news/%E2%80%98power-journey%E2%80%99-gives-back-als-community 07/23/2010 Part support group, part fundraising effort, this group seeks to fuel ALS research while offering comfort to those who are coping with the disease. http://quest.mda.org/news/utah-researchers-seek-cmd-families 07/22/2010 Families affected by Ullrich congenital MD or Bethlem myopathy are invited to help with data collection at the University of Utah. http://quest.mda.org/news/nonwalkers-sma-high-risk-weight-gain 07/22/2010 Study finds that children and adults with SMA who can't walk but otherwise have good motor function are at risk for becoming overweight. http://quest.mda.org/news/antisense-treatment-restores-full-length-smn-sma-mice 07/14/2010 Mice with a disease resembling type 3 SMA received infusions directly into the brain of a type of molecule known as a synthetic antisense oligonucleotide. http://alsn.mda.org/news/mice-and-men-als-tdi-discusses-als-research-animals 7/6/2010 Animal models play a key role in ALS research, but their usage can be tricky, said ALS TDI in its recent public webinar. http://quest.mda.org/news/dmd-gene-repair-strategy-takes-big-step-forward 06/30/2010 Researchers report making a 10-fold improvement in a technology that permanently repairs flawed dystrophin genes, giving a real boost to this therapeutic strategy. http://alsn.mda.org/news/participants-sought-three-als-trials 06/24/2010 ALS researchers are seeking participants for studies on the value of high-fat, high calorie diets; biological indicators called "biomarkers"; and the antibiotic ceftriaxone. http://quest.mda.org/news/lumizyme-now-commercially-available-pompe 06/22/2010 "Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval. http://quest.mda.org/news/first-us-exon-skipping-trial-opens 06/18/2010 A study of exon-skipping drug GSK2402968 in nonambulatory subjects with DMD has opened at Nationwide Children's Hospital in Ohio. http://quest.mda.org/news/its-myasthenia-gravis-awareness-month 06/17/2010 A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder. http://alsn.mda.org/news/flip-switch 06/15/2010 An experimental drug targets the immune system in ALS, switching cells from 'attack' to 'protective' mode. http://alsn.mda.org/news/als-ceftriaxone-trial-still-open 06/11/2010 A 600-person trial of intravenous ceftriaxone in ALS is still looking for participants at 53 North American sites. http://quest.mda.org/news/opmd-cystamine-strengthens-muscles-mice 06/09/2010 Mice with an OPMD-like disease treated with cystamine showed improved strength and muscle function and fewer cellular abnormalities. http://www.mda.org/news/100604fda-nih-mtg.html 06/04/2010 We are pleased to announce the upcoming FDA and NIH co-sponsored meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases, taking place on the 27th and 28th September, 2010 in Washington DC, USA. http://quest.mda.org/news/dmd-trial-avi4658-increased-dystrophin-production 06/02/2010 Further analysis of data from a trial of the exon-skipping drug AVI4658 showed some participants had greatly increased dystrophin production. http://alsn.mda.org/news/als-tdi-changes-works 06/01/2010 ALS TDI's May 2010 research update addressed changes: a new mouse model, new projects, a paradigm shift and a model for faster drug development. http://quest.mda.org/news/fa-research-idebenone-strikes-out-again 05/28/2010 Another trial of idebenone shows that, although safe and well tolerated, the substance doesn't lead to significant improvement in Friedreich's ataxia http://quest.mda.org/news/pompe-disease-fda-approves-lumizyme 05/25/2010 The Food and Drug Administration has OK'ed the first U.S. treatment specifically for late-onset Pompe disease http://quest.mda.org/news/podcasts-focus-latest-neuromuscular-research 05/24/2010 "This Month in Muscular Dystrophy" is the subject of a new audio series from Nationwide Children's Hospital in Columbus, Ohio. http://quest.mda.org/news/gene-therapy-success-ibm 05/21/2010 A multicenter research team has used gene therapy to improve muscle function in a single study participant with hereditary inclusion-body myositis. http://alsn.mda.org/news/sod1-versus-other-als-apples-and-oranges 05/19/2010 New research suggests non-SOD1 familial ALS and sporadic ALS may share common features — but that SOD1 familial ALS may be different. http://quest.mda.org/news/dmd-research-new-funding-imaging-studies 05/17/2010 Could MRI be better than muscle biopsy in measuring disease progression and therapy effectiveness in Duchenne muscular dystrophy? http://quest.mda.org/news/mtm-unlocking-muscle-fibers-myotubularin 05/05/2010 A new strategy may allow myotubularin protein to enter muscle, with implications for X-linked myotubular myopathy and other diseases. http://www.mda.org/research/100427_ua_research_grant.html 04/27/2010 The director of the University of Arizona’s Department of Animal Sciences, Ronald E. Allen, Ph.D., is one of 42 medical researchers and their labs who have been awarded more than $21 million in grants by MDA to advance critical neuromuscular research in 2010. http://quest.mda.org/news/new-tools-trials-children-cmt 04/23/2010 Two new pediatric scales measuring physical functioning and quality of life are good news for future clinical trials in Charcot-Marie-Tooth. http://quest.mda.org/news/curcumin-helps-mice-cmt-disease 04/21/2010 Curcumin (which makes the spice turmeric yellow) benefited mice with a disease resembling type 1B Charcot-Marie-Tooth. http://quest.mda.org/news/nt3-gene-therapy-cmt1a-benefits-mice 04/21/2010 After a single injection, mice with a disease resembling type 1A Charcot-Marie-Tooth showed improved strength and nerve signaling. http://quest.mda.org/news/caution-immune-response-seen-dmd-gene-therapy 04/21/2010 An unexpected immune response to dystrophin has researchers proceeding cautiously; the finding changes how future gene therapy trials will be conducted. http://quest.mda.org/news/dmdbmd-taking-closer-look-ataluren 04/19/2010 Trial participants receiving a low dose of ataluren did better than those receiving a placebo or high dose, additional analysis reveals. http://quest.mda.org/news/luring-away-myostatin-can-boost-muscle-size 04/16/2010 Tests of the myostatin inhibitor ACE031 show the engineered protein can safely increase muscle size, a finding with potential application for many forms of neuromuscular disease. http://quest.mda.org/news/more-good-news-about-exon-skipping 04/15/2010 A 24-person trial of intravenous AVI4658, an experimental exon skipping construct for DMD, resulted in dystrophin production and appears safe. http://quest.mda.org/news/narrowing-down-cmt-subtypes 04/15/2010 Investigators from Wayne State University found that five genetic subtypes of Charcot-Marie-Tooth disease accounted for more than 99 percent of diagnoses. http://quest.mda.org/news/smn-gene-transfer-benefits-mice 04/15/2010 British researchers report injections of SMN genes significantly increased life span in mice with a disease resembling spinal muscular atrophy. http://quest.mda.org/news/gentamicin-shows-mixed-results-dmd 04/15/2010 The drug gentamicin increased dystrophin levels in boys with DMD due to a nonsense-mutation, but didn't improve strength at the doses used. http://quest.mda.org/news/progress-exon-skipping-dmd 04/15/2010 Investigators presented intriguing data from a trial of an experimental exon skipping drug for Duchenne muscular dystrophy, at the 2010 American Academy of Neurology meeting. http://www.mda.org/research/100415cmt-grant.html 04/15/2010 Forty-two medical researchers and their labs in the U.S., Australia and Canada have been awarded more than $21 million (A$22,659,000) in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multiyear awards to be dispersed over the next three years. http://www.mda.org/research/100415mmd-grant.html 04/15/2010 Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multiyear awards to be dispersed over the next three years. http://quest.mda.org/news/als-research-zenvia-improves-emotional-symptoms-als 04/13/2010 The experimental drug Zenvia continued to show benefit in a 12-week extension study in people with "pseudobulbar affect" (uncontrolled emotional expression). http://quest.mda.org/news/mda-study-shows-lithium-not-helpful-als 04/13/2010 Despite promising results from an Italian pilot study, follow-up trials of lithium in ALS have failed to show any benefits for the drug. http://www.als-mda.org/research/news/100408john-hopkins-als-grants.html 04/08/2010 Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multiyear awards to be dispersed over the next three years. http://quest.mda.org/news/mda-awards-two-new-fshd-grants 04/05/2010 Innovative research aims to deepen understanding of the molecular cause of FSHD, with the goal of developing therapies. http://www.mda.org/research/100405_fsh.html 04/05/2010 The Muscular Dystrophy Association (MDA), headquartered in Tucson, Ariz., and Friends of FSH Research (FFSHR), based in Kirkland, Wash., today jointly awarded a two-year, $200,000 grant to Joel Chamberlain, research assistant professor of medical genetics at the University of Washington. http://quest.mda.org/news/familial-als-research-update 04/02/2010 Scientists know more about familial ALS (FALS) than sporadic ALS; FALS research may have application for both forms of the disease, however. http://www.mda.org/research/100331iowa-u.html 03/31/2010 Forty-two medical researchers and their labs have been awarded over $21 million in grants from the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multi-year awards to be dispersed over the next three years. http://www.mda.org/research/100331stjoes.html 03/31/2010 Forty-two medical researchers and their labs have been awarded over $21 million by the Muscular Dystrophy Association to continue critical neuromuscular research. Many of the grants are multi-year awards to be dispersed over the next three years. http://quest.mda.org/news/als-research-disconnecting-immune-system 03/29/2010 ALS TDI investigators have found that disrupting part of the immune system slows disease progression in mice with an ALS-like disease http://quest.mda.org/news/tadalafil-trial-becker-md 03/24/2010 The vasodilating drug tadalafil (Cialis) will be tested in men with Becker muscular dystrophy to see if it improves blood flow to forearm muscles http://quest.mda.org/news/gentamicin-trial-dmd-shows-mixed-results 03/23/2010 Intravenous gentamicin increased dystrophin production in six of 12 boys with nonsense-mutation DMD but failed to improve strength or function at the doses used http://quest.mda.org/news/dmd-research-potential-heart-saver 03/22/2010 A compound that seals cell membranes stopped progression of heart degeneration in dogs with a DMD-like disease http://quest.mda.org/news/lumizyme-horizon 03/19/2010 Commercial approval for Lumizyme, a treatment for Pompe disease, is expected in June; an early-access program exists for severely affected adults http://quest.mda.org/news/sma-research-saving-shortened-smn-protein 03/17/2010 Preserving a shortened version of the SMN protein rescued SMN-deficient cells, opening the door to a possible new therapeutic strategy http://quest.mda.org/news/gene-therapy-book-mda-grantee 03/08/2010 'Muscle Gene Therapy' brings together the latest research in this promising field. http://quest.mda.org/news/als-sod1-trial-%E2%80%98watershed-moment%E2%80%99 03/05/2010 Human testing has begun of ISIS-SOD1-Rx, a compound designed to block production of a toxic protein in people with the SOD1-related form of familial (inherited) ALS http://quest.mda.org/news/dmdbmd-research-ataluren-results-disappointing 03/05/2010 The experimental drug ataluren, developed to overcome nonsense mutations in Duchenne and Becker MD, did not meet its primary end point in a large-scale human trial. http://quest.mda.org/news/gene-therapy-rescues-mice-sma 03/01/2010 A research team reports "unprecedented" improvement in newborn SMA-affected mice that received gene therapy via intravenous injection. http://quest.mda.org/news/quicker-trip-%E2%80%98microscope-marketplace%E2%80%99 02/27/2010 Two federal agencies, the NIH and FDA, are cooperating in an attempt to reduce the time it takes to approve new drugs. http://quest.mda.org/news/sma-therapeutics-changing-code 02/27/2010 Researchers have demonstrated that a technique called trans-splicing can lessen the severity of symptoms and increase life span in mice with SMA. http://quest.mda.org/news/building-new-muscle-requires-controlled-damage 02/23/2010 MDA-supported scientists in Ottawa have found that controlled damage to DNA is required before immature muscle cells can mature into muscle fibers. http://quest.mda.org/news/sma-research-strengthening-junctions 02/22/2010 MDA-supported researchers in Ottawa have identified a biological pathway that could become a new treatment avenue in spinal muscular atrophy (SMA). http://quest.mda.org/news/causative-gene-mutations-idd-two-muscle-diseases 02/18/2010 Different mutations in a gene called ANO5 cause type 2L LGMD (LGMD2L) and type 3 Miyoshi myopathy. http://quest.mda.org/news/cardiomyopathy-becker-md-0 02/12/2010 Researchers have found that specific mutations in the dystrophin gene correlate with earlier or later onset of cardiac muscle degeneration. http://quest.mda.org/news/cmd-form-mm-qualify-speedy-decision-ss-benefits 02/11/2010 Social Security has added four forms of congenital muscular dystrophy and Leigh syndrome to its Compassionate Allowances list of disabilities that qualify for a quick decision on benefits. http://quest.mda.org/news/new-muscle-stem-cell-found-mice 02/09/2010 Researchers in France have identified a new type of muscle stem cell that appears capable of generating and repairing muscle and reproducing itself. http://quest.mda.org/news/dmd-bmd-tests-start-experimental-utrophin-drug 02/09/2010 Human testing has begun of BMN195, a compound designed to increase the body’s production of utrophin, which may shelter fragile muscle cells in DMD and BMD. http://quest.mda.org/news/potassium-channel-mutations-underlie-tpp 02/03/2010 Mutations in a gene for a newly discovered potassium channel can cause some cases of thyrotoxic hypokalemic periodic paralysis (TPP). http://quest.mda.org/news/mmd1-lost-proteins-roles-revealed 02/02/2010 Loss of a protein known as MBNL2 may be responsible for the weakness and muscle atrophy seen in type 1 myotonic dystrophy. http://quest.mda.org/news/als-tdi-full-speed-ahead 01/28/2010 ALS TDI's first-quarter 2010 research update can be summed up with one word: momentum. http://quest.mda.org/news/parent-survey-dmd-bmd-cmd-and-sma 01/21/2010 An online survey aims to reduce the time it takes parents to get an accurate diagnosis for their children. http://www.mda.org/research/100119ataluren-ptc-therapeutics.html 01/19/2010 PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently. http://quest.mda.org/news/dmd-bmd-trial-ataluren-nonwalkers 01/19/2010 PTC Therapeutics, with support from MDA, is testing ataluren in boys with DMD or BMD who have nonsense mutations and are no longer walking. http://quest.mda.org/news/gene-links-rare-cmt-sma-forms 01/11/2010 Researchers have identified different mutations in a gene called TRPV4 as causing type 2C CMT and two rare forms of SMA. http://quest.mda.org/news/opmd-toxic-clumps-not-only-cause 01/07/2010 Investigators have found that a loss of function of the PABPN1 protein, in addition to clump formation, may contribute to OPMD. http://quest.mda.org/news/mda-awards-21-million-research 01/06/2010 New MDA grants recently were approved for several forms of muscular dystrophy, ALS, Friedreich’s ataxia, myasthenia gravis and other diseases in MDA's program. http://www.als-mda.org/research/news/100106als-tdi-mda-partnership.html 01/06/2010 Buoyed by the extraordinary progress being made by the ALS Therapy Development Institute, the Muscular Dystrophy Association today announced a new milestone-driven grant of $2.5 million, adding to the $18 million MDA already has invested with ALS TDI - the world’s only non profit research center focused exclusively on developing treatments for amyotrophic lateral sclerosis ALS.