Living with a rare disease brings unique challenges, not only for the person diagnosed but also for their families and caregivers. Getting the right diagnosis can take years, and treatment options are often limited.
One example is myasthenia gravis, a chronic autoantibody disease in which harmful antibodies disrupt nerve-to-muscle communication, leading to severe and unpredictable muscle weakness. In its more severe form, generalized myasthenia gravis (gMG), this weakness can become widespread and unpredictable, impacting essential daily functions such as speaking, swallowing and even breathing.
Standard therapies such as oral corticosteroids and broad immunosuppressants can help reduce gMG symptoms, but they also suppress the entire immune system, increasing vulnerability to infections and leading to long-term health concerns such as heart disease, weight gain and gastrointestinal complications.
A new treatment from Johnson & Johnson takes a more targeted approach. It blocks a protein called
neonatal Fc receptor, which helps lower the level of immunoglobulin G, one of the root causes of autoantibody diseases—potentially without impacting other immune functions. First approved by the FDA earlier this year, the therapy has since received additional authorizations and is under regulatory review in countries around the world.
On the horizon: This treatment is being studied across multiple autoantibody diseases, including
idiopathic inflammatory myopathies, chronic inflammatory demyelinating polyneuropathy,
warm autoimmune hemolytic anemia and
Sjögren’s disease. It’s also being investigated in
hemolytic disease of the fetus and newborn and
fetal and neonatal alloimmune thrombocytopenia,
alloantibody diseases in which maternal antibodies attack fetal or newborn cells—and where the treatment has the potential to revolutionize care.