//www.koonmotors.com/news/health_medicine/muscular_dystrophy/ Medical research on muscular dystrophy and myopathy. Read about the promise of stem cell research for muscular dystrophy patients, and successful treatments tested in dogs. en-us Wed, 06 Sep 2023 14:46:38 EDT Wed, 06 Sep 2023 14:46:38 EDT 60 //www.koonmotors.com/images/scidaily-logo-rss.png//www.koonmotors.com/news/health_medicine/muscular_dystrophy/ For more science news, visit ScienceDaily. //www.koonmotors.com/releases/2023/08/230824111922.htm Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein function in induced pluripotent stem cells derived from DMD patients. The approach worked by removing large sections of the dystrophin gene, allowing the cells to skip faulty or misaligned sections of the genetic code. This yields truncated but still functional proteins for a wide variety of mutation patterns associated with DMD. Thu, 24 Aug 2023 11:19:22 EDT //www.koonmotors.com/releases/2023/08/230824111922.htm //www.koonmotors.com/releases/2023/08/230816114121.htm A new method allows large quantities of muscle stem cells to be safely obtained in cell culture. This provides a potential for treating patients with muscle diseases -- and for those who would like to eat meat, but don't want to kill animals. Wed, 16 Aug 2023 11:41:21 EDT //www.koonmotors.com/releases/2023/08/230816114121.htm //www.koonmotors.com/releases/2023/07/230731151434.htm The research team administered AP39 to some worms from birth, and to others after reaching adulthood. They found that this compound improved the integrity of mitochondria -- the 'power house' of cells, which produces our cells' energy, and kept the worms' muscles active and moving, even well into old age, and when given mid-way through their life-course. The research concludes that targeting H2S specifically to the energy-generating machinery of cells (mitochondria) could one day be used as a healthy aging therapeutic. Mon, 31 Jul 2023 15:14:34 EDT //www.koonmotors.com/releases/2023/07/230731151434.htm //www.koonmotors.com/releases/2023/05/230524181848.htm A research team has discovered that by manipulating a certain protein in the immune system they can slow down disease progression and improve muscle function in Duchenne muscular dystrophy. Wed, 24 May 2023 18:18:48 EDT //www.koonmotors.com/releases/2023/05/230524181848.htm //www.koonmotors.com/releases/2023/04/230413154253.htm Investigators recently used a targeted drug to restore muscle strength and correct myotonia in mice with myotonic dystrophy. Thu, 13 Apr 2023 15:42:53 EDT //www.koonmotors.com/releases/2023/04/230413154253.htm //www.koonmotors.com/releases/2023/02/230227132657.htm New research has uncovered a novel link between the Duchenne muscular dystrophy (DMD) gene and cancer. Mon, 27 Feb 2023 13:26:57 EST //www.koonmotors.com/releases/2023/02/230227132657.htm //www.koonmotors.com/releases/2022/12/221219124256.htm Therapies to target neuromuscular disorders affecting million of people worldwide are on the horizon thanks to research at the Montreal Clinical Research Institute of Montreal. Fusion of myoblasts, the stem cells responsible for the formation of skeletal muscles, could allow the repair of muscles damaged by diseases such as muscular dystrophy. Mon, 19 Dec 2022 12:42:56 EST //www.koonmotors.com/releases/2022/12/221219124256.htm //www.koonmotors.com/releases/2022/12/221212140646.htm Fukuyama congenital muscular dystrophy (FCMD) is the second most common form of childhood muscular dystrophy in Japan. The disease manifests itself in the form of a severe neuromuscular disorder. One form of FCMD is caused by a genetic abnormality in the 'fukutin' (FKTN) gene. The genetic anomaly blocks the chemical glycosylation of a biologically important protein. Using RNA interference, Japanese researchers restored the normal biological function of FKTN in patient-derived cells, offering hope for therapy. Mon, 12 Dec 2022 14:06:46 EST //www.koonmotors.com/releases/2022/12/221212140646.htm //www.koonmotors.com/releases/2022/09/220913140852.htm Researchers report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne muscular dystrophy (DMD). Tue, 13 Sep 2022 14:08:52 EDT //www.koonmotors.com/releases/2022/09/220913140852.htm //www.koonmotors.com/releases/2022/05/220504135635.htm New research recommends daily steroid doses for children with Duchenne muscular dystrophy, marking a significant change in how the disease is treated. Wed, 04 May 2022 13:56:35 EDT //www.koonmotors.com/releases/2022/05/220504135635.htm //www.koonmotors.com/releases/2022/04/220420151643.htm In proof-of-concept experiments, scientists say they have successfully cultivated human muscle stem cells capable of renewing themselves and repairing muscle tissue damage in mice, potentially advancing efforts to treat muscle injuries and muscle-wasting disorders in people. Wed, 20 Apr 2022 15:16:43 EDT //www.koonmotors.com/releases/2022/04/220420151643.htm //www.koonmotors.com/releases/2022/04/220413161733.htm A cell therapy stabilizes weakened muscles -- including the heart muscle -- in Duchenne muscular dystrophy patients, a new study shows. Wed, 13 Apr 2022 16:17:33 EDT //www.koonmotors.com/releases/2022/04/220413161733.htm //www.koonmotors.com/releases/2022/04/220411120711.htm Patients who took part in the study saw a 1.6-kilogram increase in their muscle mass and a two per cent reduction of body fat. They were also able to walk an extra 47 meters in six minutes, when tested by researchers at the end of the 12-week trial. Eleven patients took part in the study. Mon, 11 Apr 2022 12:07:11 EDT //www.koonmotors.com/releases/2022/04/220411120711.htm //www.koonmotors.com/releases/2022/04/220401122235.htm Obese mice that were fed a high-fat diet and that received prednisone one time per week had improved exercise endurance, got stronger, increased their lean body mass and lost weight, reports a new study. The mice also had increased muscle metabolism. The once-weekly prednisone promoted nutrient uptake into the muscles. Fri, 01 Apr 2022 12:22:35 EDT //www.koonmotors.com/releases/2022/04/220401122235.htm //www.koonmotors.com/releases/2022/03/220324184652.htm A new study using neuromuscular electrical stimulation (NMES) in zebrafish found that certain activities may help strengthen muscles affected by Duchenne muscular dystrophy, a severe type of muscular dystrophy that affects young boys. The researchers designed four NMES regimens for zebrafish with a mutation that closely modeled the disease. They then put the fish into an underwater 3D printed 'gym' made up of tunnels and electrodes, and analyzed their skeletal muscles to see how they had changed. Thu, 24 Mar 2022 18:46:52 EDT //www.koonmotors.com/releases/2022/03/220324184652.htm //www.koonmotors.com/releases/2022/03/220318161432.htm Tracing the impact of a single protein, Piezo1, researchers found that restoring it in muscles affected by Duchenne muscular dystrophy could improve their ability to heal efficiently. Fri, 18 Mar 2022 16:14:32 EDT //www.koonmotors.com/releases/2022/03/220318161432.htm //www.koonmotors.com/releases/2022/03/220310185956.htm A recent clinical trial has shown that cellular therapy is safe and effective in stopping the deterioration of upper limb and heart functions in patients with late-stage Duchenne muscular dystrophy. Thu, 10 Mar 2022 18:59:56 EST //www.koonmotors.com/releases/2022/03/220310185956.htm //www.koonmotors.com/releases/2022/02/220223164606.htm A new 'cocktail' drug under development could provide an effective and economical treatment to lessen symptoms for up to 45 per cent of patients with Duchenne muscular dystrophy (DMD), a chronic muscle-wasting disease. Wed, 23 Feb 2022 16:46:06 EST //www.koonmotors.com/releases/2022/02/220223164606.htm //www.koonmotors.com/releases/2022/02/220218153028.htm Latest advance in chronopharmacology, based on mouse models, suggests that time-of-day matters when using steroids to treat muscle diseases. Fri, 18 Feb 2022 15:30:28 EST //www.koonmotors.com/releases/2022/02/220218153028.htm //www.koonmotors.com/releases/2022/01/220128141257.htm Scientists have made a link between muscular dystrophy and a group of bioactive fats, the sphingolipids, which are involved in numerous cell functions and other diseases. Fri, 28 Jan 2022 14:12:57 EST //www.koonmotors.com/releases/2022/01/220128141257.htm //www.koonmotors.com/releases/2022/01/220119135032.htm Researchers have published findings that could contribute to future therapeutics for muscle degeneration due to old age, and diseases such as cancer and muscular dystrophy. Wed, 19 Jan 2022 13:50:32 EST //www.koonmotors.com/releases/2022/01/220119135032.htm //www.koonmotors.com/releases/2022/01/220113111453.htm A newly characterized fat protein could provide the missing link to explain a rare metabolic disease while offering fresh insight into common disorders such as obesity and type 2 diabetes. Thu, 13 Jan 2022 11:14:53 EST //www.koonmotors.com/releases/2022/01/220113111453.htm //www.koonmotors.com/releases/2021/12/211220144957.htm 研究人员已经确定了所需的蛋白质for efficient regeneration of skeletal muscles after acute injury and in Duchenne Muscular Dystrophy (DMD) clearing the way for drug development for skeletal muscle diseases. Mon, 20 Dec 2021 14:49:57 EST //www.koonmotors.com/releases/2021/12/211220144957.htm //www.koonmotors.com/releases/2021/07/210706115430.htm Muscles and the resident stem cells (satellite cells) responsible for muscle regeneration retain memory of their location in the body. This positional memory was found to be based on the expression pattern of the homeobox (Hox) gene cluster, which is responsible for shaping the body during fetal life. These findings are expected to provide clues to elucidate the pathogenesis of muscle diseases like muscular dystrophy, and help develop regenerative treatments based on positional memory. Tue, 06 Jul 2021 11:54:30 EDT //www.koonmotors.com/releases/2021/07/210706115430.htm //www.koonmotors.com/releases/2021/06/210624161707.htm Researchers have developed a new approach in which specifically designed anti-inflammatory nanoparticles (NPs) that could be applied locally and selectively to chronically inflamed muscles severely affected or at more immediate risk of deterioration, and maybe difficult to reach with oral therapeutics. Thu, 24 Jun 2021 16:17:07 EDT //www.koonmotors.com/releases/2021/06/210624161707.htm //www.koonmotors.com/releases/2021/04/210414154914.htm A new study reveals how chronic inflammation promotes muscle fibrosis, which could inform the development of new therapies for patients suffering from Duchenne muscular dystrophy (DMD), a fatal muscle disease. Wed, 14 Apr 2021 15:49:14 EDT //www.koonmotors.com/releases/2021/04/210414154914.htm //www.koonmotors.com/releases/2021/03/210318161128.htm A research team has identified a chemical cocktail that enables the production of large numbers of muscle stem cells, which can self-renew and give rise to all types of skeletal muscle cells. Thu, 18 Mar 2021 16:11:28 EDT //www.koonmotors.com/releases/2021/03/210318161128.htm //www.koonmotors.com/releases/2021/01/210112125223.htm Cell-derived exosomes are effective in treating disease when mixed with the dominant protein in breast milk and given orally, a new study of laboratory mice shows. The findings could help develop new oral medications for treating patients with muscular dystrophy and heart failure. Tue, 12 Jan 2021 12:52:23 EST //www.koonmotors.com/releases/2021/01/210112125223.htm //www.koonmotors.com/releases/2020/12/201211115513.htm A muscle fiber consists of just one cell, but many nuclei. A team has now shown just how varied these nuclei are. The study can help us better understand muscle diseases such as Duchenne muscular dystrophy. Fri, 11 Dec 2020 11:55:13 EST //www.koonmotors.com/releases/2020/12/201211115513.htm //www.koonmotors.com/releases/2020/11/201130131451.htm Adding exercise to a genetic treatment for myotonic dystrophy type 1 was more effective at reversing fatigue than administering the treatment alone in a study using a mouse model of the disease. In fact, exercise alone provided some benefit whereas the genetic treatment alone did not. This study has implications for patients with fatigue due to genetics-related musculoskeletal diseases and other types of illness-induced fatigue. Mon, 30 Nov 2020 13:14:51 EST //www.koonmotors.com/releases/2020/11/201130131451.htm //www.koonmotors.com/releases/2020/11/201104085145.htm A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study suggests. The findings could help doctors develop new strategies to preserve heart function in this population, potentially extending patients' lives. Wed, 04 Nov 2020 08:51:45 EST //www.koonmotors.com/releases/2020/11/201104085145.htm //www.koonmotors.com/releases/2020/09/200915090134.htm 科学家已经证明,药物(毒品)correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition, and has far-reaching implications for the field of regenerative medicine. Tue, 15 Sep 2020 09:01:34 EDT //www.koonmotors.com/releases/2020/09/200915090134.htm //www.koonmotors.com/releases/2020/09/200911141747.htm Researchers have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered 'undruggable.' Fri, 11 Sep 2020 14:17:47 EDT //www.koonmotors.com/releases/2020/09/200911141747.htm //www.koonmotors.com/releases/2020/08/200827105948.htm Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. Researchers have found a connection between dystrophic muscles and the lymphatic system in mice with Duchenne disease. Thu, 27 Aug 2020 10:59:48 EDT //www.koonmotors.com/releases/2020/08/200827105948.htm //www.koonmotors.com/releases/2020/06/200629164141.htm Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy. Medical researchers have created and tested synthetic DNA-like molecules that interfere with the production of a toxic protein that destroys the muscles of people who have facioscapulohumeral muscular dystrophy (FSHD). Mon, 29 Jun 2020 16:41:41 EDT //www.koonmotors.com/releases/2020/06/200629164141.htm //www.koonmotors.com/releases/2020/04/200429144924.htm Scientists have taken a step closer towards developing a treatment for the long-term genetic disorder, myotonic dystrophy. Wed, 29 Apr 2020 14:49:24 EDT //www.koonmotors.com/releases/2020/04/200429144924.htm //www.koonmotors.com/releases/2020/04/200424132702.htm Children born with Duchenne muscular dystrophy have a mutation in the X-chromosome gene that would normally code for dystrophin, a protein that provides structural integrity to skeletal muscles. The loss of this protein causes severe symptoms, including deteriorating muscle strength beginning around the age of four. While there is no cure, a promising area of research has developed around the protein utrophin, which is approximately 80 percent identical to dystrophin and even takes its place early during muscle development. Fri, 24 Apr 2020 13:27:02 EDT //www.koonmotors.com/releases/2020/04/200424132702.htm //www.koonmotors.com/releases/2020/02/200224131133.htm Testing of small molecules in mouse models for Duchenne muscular dystrophy shows promise for restoration of muscle structure and function. Mon, 24 Feb 2020 13:11:33 EST //www.koonmotors.com/releases/2020/02/200224131133.htm //www.koonmotors.com/releases/2020/01/200127134851.htm Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers have developed a gene therapy that may provide permanent relief for those suffering from DMD. Mon, 27 Jan 2020 13:48:51 EST //www.koonmotors.com/releases/2020/01/200127134851.htm //www.koonmotors.com/releases/2019/12/191209102044.htm Repairing faulty genes to prevent and cure disease is something researchers have been working towards for many years. While Class 2 CRISPR systems show great promise as gene editing tools in human cells, a research team has now demonstrated that a Cas3-based Class 1 CRISPR system may provide a more efficient and safer alternative, carrying out successful repair of a gene mutation responsible for Duchenne muscular dystrophy in patient-derived cells. Mon, 09 Dec 2019 10:20:44 EST //www.koonmotors.com/releases/2019/12/191209102044.htm //www.koonmotors.com/releases/2019/09/190925133645.htm A new therapeutic being tested is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD). The treatment -- a cocktail of DNA-like molecules -- results in dramatic regrowth of a protein called dystrophin, which acts as a support beam to keep muscles strong. The protein is virtually absent in those with DMD. Wed, 25 Sep 2019 13:36:45 EDT //www.koonmotors.com/releases/2019/09/190925133645.htm //www.koonmotors.com/releases/2019/09/190924080032.htm A new clinical trial found a cost-effective generic drug works just as well as a more expensive drug in preserving heart function in boys with Duchenne muscular dystrophy. Tue, 24 Sep 2019 08:00:32 EDT //www.koonmotors.com/releases/2019/09/190924080032.htm //www.koonmotors.com/releases/2019/09/190917115430.htm Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder. Tue, 17 Sep 2019 11:54:30 EDT //www.koonmotors.com/releases/2019/09/190917115430.htm //www.koonmotors.com/releases/2019/07/190718112548.htm A team has identified a gene that could make immunotherapy treatments, specifically checkpoint inhibitors, work for a wider variety of cancer patients. The study found that when the DUX4 gene is expressed in cancer cells, it can prevent the cancer from being recognized and destroyed by the immune system. Thu, 18 Jul 2019 11:25:48 EDT //www.koonmotors.com/releases/2019/07/190718112548.htm //www.koonmotors.com/releases/2019/07/190709122031.htm Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe progressive deterioration are needed to extend lives and improve quality of life. Results of three clinical trials using eteplirsen, an exon-skipping antisense oligonucleotide, show promising results. Tue, 09 Jul 2019 12:20:31 EDT //www.koonmotors.com/releases/2019/07/190709122031.htm //www.koonmotors.com/releases/2019/06/190614125838.htm Researchers found that the protein sarcospan can play a major role in combating heart failure in patients with Duchenne muscular dystrophy. Fri, 14 Jun 2019 12:58:38 EDT //www.koonmotors.com/releases/2019/06/190614125838.htm //www.koonmotors.com/releases/2019/06/190607110516.htm Muscle decline caused by ageing and certain diseases could be dramatically slowed by stopping a chain reaction that damages cells, new research shows. Fri, 07 Jun 2019 11:05:16 EDT //www.koonmotors.com/releases/2019/06/190607110516.htm //www.koonmotors.com/releases/2019/06/190603124717.htm Researchers have found that lithium improves muscle size and strength in mice with a rare form of muscular dystrophy that causes weakness in the shoulders and hips. The findings could lead to a drug for the disabling condition. Mon, 03 Jun 2019 12:47:17 EDT //www.koonmotors.com/releases/2019/06/190603124717.htm //www.koonmotors.com/releases/2019/04/190417084510.htm Scientists have uncovered a molecular signaling pathway involving Stat3 and Fam3a proteins that regulates how muscle stem cells decide whether to self-renew or differentiate -- an insight that could lead to muscle-boosting therapeutics for muscular dystrophies or age-related muscle decline. Wed, 17 Apr 2019 08:45:10 EDT //www.koonmotors.com/releases/2019/04/190417084510.htm //www.koonmotors.com/releases/2019/04/190410120021.htm Scientists have developed a strategy for editing and repairing a particular type of genetic mutation associated with microduplications using CRISPR/Cas9 and a seldom-used DNA repair pathway. This approach to programmable gene editing overcomes prior inefficiencies in gene correction. Wed, 10 Apr 2019 12:00:21 EDT //www.koonmotors.com/releases/2019/04/190410120021.htm //www.koonmotors.com/releases/2019/04/190401105311.htm The compound called Cugamycin works by recognizing toxic RNA repeats and destroying the garbled gene transcript. Mon, 01 Apr 2019 10:53:11 EDT //www.koonmotors.com/releases/2019/04/190401105311.htm //www.koonmotors.com/releases/2019/03/190312123640.htm A research team how a type of protein that is embedded in the inner nuclear membrane clears out of the system once it has served its purpose. Tue, 12 Mar 2019 12:36:40 EDT //www.koonmotors.com/releases/2019/03/190312123640.htm //www.koonmotors.com/releases/2019/03/190306171244.htm Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases. Wed, 06 Mar 2019 17:12:44 EST //www.koonmotors.com/releases/2019/03/190306171244.htm //www.koonmotors.com/releases/2019/02/190227111135.htm By sequencing the entire genomes of tumor cells from six people with a rare cancer of the nose and sinus cavity, researchers report they unexpectedly found the same genetic change -- one in a gene involved in muscle formation -- in five of the tumors. Wed, 27 Feb 2019 11:11:35 EST //www.koonmotors.com/releases/2019/02/190227111135.htm //www.koonmotors.com/releases/2019/02/190221130242.htm Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects. Thu, 21 Feb 2019 13:02:42 EST //www.koonmotors.com/releases/2019/02/190221130242.htm //www.koonmotors.com/releases/2019/02/190213172310.htm The University of Minnesota Medical School continues its legacy of advancing cell replacement therapies with a scientific breakthrough that highlights the promise of cell therapies for muscular dystrophy. Wed, 13 Feb 2019 17:23:10 EST //www.koonmotors.com/releases/2019/02/190213172310.htm //www.koonmotors.com/releases/2019/02/190211095455.htm Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects. Eplerenone, a heart failure drug, is used in older patients to treat cardiomyopathy, a leading cause of mortality for people with DMD. A new medicine under development appears to combine the beneficial effects of these drugs for the heart and muscle while also showing improved safety in experimental models. Mon, 11 Feb 2019 09:54:55 EST //www.koonmotors.com/releases/2019/02/190211095455.htm //www.koonmotors.com/releases/2019/02/190201114120.htm Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment gene. Fri, 01 Feb 2019 11:41:20 EST //www.koonmotors.com/releases/2019/02/190201114120.htm //www.koonmotors.com/releases/2019/01/190131113918.htm Researchers have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. The team restored muscle stem cell function that is impaired in Duchenne muscular dystrophy, resulting in efficient regeneration of the muscle and preventing the progressive loss of muscle strength characteristic of the disease. Thu, 31 Jan 2019 11:39:18 EST //www.koonmotors.com/releases/2019/01/190131113918.htm //www.koonmotors.com/releases/2018/12/181212200809.htm To help patients with muscle disorders, scientists have engineered a new stem cell line to study the conversion of stem cells into muscle. Wed, 12 Dec 2018 20:08:09 EST //www.koonmotors.com/releases/2018/12/181212200809.htm