In a recent study titled “Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes,” a research team from the Department of Pediatric Neurology at the Catholic University in Rome, Italy examined the changes at 3 years follow-up in a 6-minute walk test…
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The Muscular Dystrophy Association (MDA) recently announced that MDA clinics are offering genetic testing for those with limb-girdle muscular dystrophy (LGMD) thanks to a grant from Sanofi company Genzyme in collaboration with Emory Genetics Laboratory. This represents a major step in diagnostic advances that will help those with the disease and their respective clinicians so they can find…
Patients in Indiana who suffer from terminal diseases such as muscular dystrophy may now have access to experimental drugs that have not yet been approved by the U.S. Food and Drug Administration (FDA), but are thought to be beneficial in reducing the burden of the disease or improving patients’…
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to naproxcinod, an investigational treatment being developed by Nicox S.A. for patients with Duchenne muscular dystrophy (DMD). The designation is given to drugs or biological products that address rare conditions and is meant…
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Dilated Cardiomyopathy Using Induced Pluripotent Stem Cells Derived From Duchenne Muscular Dystrophy
A recent study published in the journal Disease Models & Mechanisms uncovered important underlying molecular mechanisms associated with cardiomyopathy in Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD) is caused by mutations in the dystrophin gene (DMD), and characterized by progressive weakness in skeletal and cardiac muscles.
PhaseBio Pharmaceuticals, Inc., a leading company in biopolymer-based drugs developing treatments to address metabolic and specific cardiopulmonary disorders, recently announced that a $40 million financing round led by AstraZeneca has been closed. This investment from AstraZeneca, Hatteras Venture Partners, New Enterprise Associates and Johnson & Johnson Innovation – JJDC and Fletcher Spaght Ventures will serve…
Santhera Pharmaceuticals‘ Principal Investigator Gunnar M. Buyse will be presenting Phase III data on Idebenone (Raxone®/Catena®) for the treatment of Duchenne Muscular Dystrophy (DMD) at the Annual Meeting of the American Academy of Neurology (AAN). Buyse’s oral presentation entitled, “Idebenone Reduces Loss of Respiratory Function in Duchenne Muscular Dystrophy-Outcome…
Findings from a recent case report published online in the journal Respiratory Medicine Case Reports suggest that patients with Facioscapulohumeral muscular dystrophy (FSHD) with nocturnal hypoventilation should have their diaphragm assessed, because nocturnal hypoventilation can be treated effectively by non-invasive ventilation. Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal…
Israeli biotech company BioBlast Pharma Ltd. has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application and is now authorized to advance development of lead product Cabaletta. The chemical chaperone is expected to become a therapeutic option for the…
The Muscular Dystrophy Association’s president and CEO opened its 2015 Scientific Conference in Washington this week by underscoring the organization’s main focus to accelerate discoveries of Duchenne muscular dystrophy (DMD) treatments for the families they serve. Steven M. Derks stated in his keynote address that the MDA has an audacious five-year plan for…
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