homozygous familial hypercholesterolemia

Cholesterol drug misbranded, company pleads guilty
Cholesterol drug misbranded, company pleads guiltyPharma maker pleads guilty that one of its major drugs was misbranded in a federal court case brought by FDA and the US Department of Justice.
Patient access to orphan drugs faces new set of challengesIn 1983, the United States enacted the Orphan Drug Act (ODA). An analogous law was passed in Europe in 2000. Both pieces of legislation are considered major successes in terms of spurring the development of orphan drugs. To illustrate, in the decade prior to 1983 only 34 orphan products were marketed, whereas in the past year alone 9 orphan drugs were launched. In the past 5 years, 39 orphan drugs were launched in the US across numerous therapeutic categories, including multiple myeloma, chronic myeloid leukemia, metastatic non-small cell lung cancer, hemophilia, tuberculosis, homozygous familial hypercholesterolemia, and cystic fibrosis.1