Orphan Drugs Homework

Orphan Drugs

Orphan drugs are considered adequate drugs for certain diseases and conditions which affect a very small number of individuals in the United States, and are considered rare in the US. Since so few people are affected by one rare disease or condition, a pharmaceutical company which develops an orphan drug may reasonably expect the drug to generate in smaller scales in comparison to the cost of developing the drug and may incur a financial loss. There is reason to believe that some promising orphan drugs will not be developed unless changes are made to the federal laws to reduce the costs of developing these drugs. Some rare diseases and conditions include: ALS (Lou Gehrig’s disease), Tourette syndrome, Huntington’s disease, and muscular dystrophy.

Charcot-Marie-Tooth disease (CMT) is a group of genetic nerve disorders. I was named after the three doctors who first identified it. The disorder affects about 1 in 2,500 people. CMT affects your peripheral nerves, the nerves that carry movement and sensation signals between your brain, spinal cord, and the rest of your body. Some of the early symptoms are foot problems such as high arches or hammertoes, as it progresses your lower legs may become weaker. Later, your hands may also start to become weak. There is no cure for the disorder. Type 1 is the most common of the group, but it is not life threatening. It almost never affects the brain. (RS) baclofen, naltrexone, and D-sorbitol are the generic names of the new orphan drug that has been designated for the treatment of CMT type 1A. Although the three drugs are unrelated to the disorder, it has seemed to work. This new drug doesn’t have a trade name, and has not yet been approved by the FDA for Orphan Indication. Baclofen is a clinically used drug that treats spasticity, which is a syndrome characterized by excessive contraction of the muscles and hyperflexia in the central nervous system. Naltrexone is a drug used to manage opioid and alcohol...