Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…
In the United States, Breakthrough Therapy designation is granted by the U.S. Food and Drug Administration (FDA) to expedite drug development and review. This designation is granted to therapies that…
In the United States, the FDA grants Breakthrough Therapy designation to drugs intended to treat serious and life-threatening illnesses, and/or drugs which show significant improvement over available therapies. After receiving…
The China Center for Drug Evaluation (CDE) has recently granted Harbour BioMed the Breakthrough Therapy designation for batoclimab, a treatment for adults with myasthenia gravis (MG). This designation was created…
According to a recent press release, EspeRare received Breakthrough Therapy Designation for its investigational therapy ER-004, designed for the prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). Ultimately, this…
As originally reported in Porphyria News, rare diseases have historically been neglected by traditional medicine; their obscure and often genetic nature made them lack priority, stuck behind a long list…
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