A phase 2 clinical trial of the drug rituximab (Rituxan) in adults with myasthenia gravis (MG) is now open at 15 U.S. centers with plans for additional sites.
Rituximab suppresses a specific part of the body's immune system and is approved by the U.S. Food and Drug Administration (FDA) to treat rheumatoid arthritis and other disorders.
The investigational oral drug ataluren, in development to treat Duchenne muscular dystrophy (DMD) resulting from a specific type of genetic mutation, has received conditional approval in the European Union (EU). This designation means patients can receive it in the EU and EU-associated countries while additional studies are being conducted.
California-based Isis Pharmaceuticals has announced the opening of a phase 3 clinical trial of its experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA), a muscle-weakening disease that results from loss of nerve cells in the spinal cord.
ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.
Matthew Martin and Amanda Hall have more than a few things in common. They’re roughly the same age — Martin is 28; Hall is 30. They both call the state of Texas home — Martin in Rowlett; Hall in Glenrose. Then there’s the fact that each lives with a neuromuscular disease — Martin was diagnosed with Duchenne muscular dystrophy (DMD) at the age of 8; Hall with congenital myopathy in her early 20s.
Whether you are newly diagnosed or have been living with a neuromuscular disease for years, there is arguably one guiding principle that is most important when it comes to making decisions about your care and quality of life: You are in charge.
And with good reason.