A committee convened by the American Academy of Neurology has published new recommendations for doctors who see children with congenital muscular dystrophy
This spring, the American Academy of Neurology (AAN) released a guideline for the diagnosis and care of children with congenital muscular dystrophy (CMD), a group of early-onset, genetic disorders that can involve skeletal (including respiratory) muscle weakness, heart problems, brain and eye abnormalities, and contractures (frozen joints).
An early-onset disease with many genetic causes
Women with neuromuscular disorders need the same gynecologic care as other women — and sometimes more
Katrina Gossett knows the importance of gynecologic (pelvic) exams, but she is not eager to repeat her last exam experience.
Katrina Gossett, 29, has only had one gynecologic (pelvic) exam in her life, and it’s not an experience she’s eager to repeat.
Through its fundraising, advocacy, health care services and support network, MDA helps make possible the everyday successes of its families and the lifesaving work of its researchers
For people fighting neuromuscular disease,“progress” is a word that describes encouraging advances in the search for treatments and cures. But it’s also a word that evokes the forward-looking perspectives and remarkable life journeys of those living with these diseases who meet extraordinary challenges.
Facing a neuromuscular disease diagnosis can be devastating, overwhelming and confusing, but empowering yourself to take charge of your care or your child's care is more important than ever
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.
Drug development consultant Abby Bronson says early access to a new drug can jeopardize its full approval and long-term availability
Abby Bronson is the Duchenne muscular dystrophy program manager at Children's National Medical Center in Washington, D.C., where she works with various stakeholders to further therapeutic development for this disease. Bronson has a master's degree in business administration from the University of Pennsylvania and has managed new product development and marketing at biotechnology and...
Bioethicist Arthur Caplan says early access to drugs should not be determined by drug companies but by independent advisory boards
Arthur Caplan is the William F. and Virginia Connolly Mitty Professor and founding head of the Division of Bioethics at New York University Langone Medical Center in New York City. He holds a doctoral degree in the history and philosophy of science from Columbia University, has authored or edited 32 books and more than 600 papers, and has served on national and international committees as an...
FDA staffer Richard Klein says people often underestimate the risks and overestimate the benefits of taking medications that have not been through the full FDA approval process
Richard Klein is the director of the Patient Liaison Program at the Office of Health and Constituent Affairs at the U.S. Food and Drug Administration (FDA).
Q: What is the most common misconception that patient communities have about the FDA's accelerated approval mechanism?