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Rare diseases impact very small portions of a population, which means they were often overlooked in the past. Often, these diseases attract less funding, stunting research and development related to treatments and cures. The Orphan Drug Act was passed in the United States in 1983 to help address this issue by incentivizing drug research to treat so-called orphan diseases. The act defines rare diseases as conditions that impact fewer than 200,000 people in the United States. In the 40 years after the Orphan Drug Act was passed, approved treatments for rare diseases grew from 38 to more than 800. Those numbers include some products from Sentynl Therapeutics, Inc. Challenges with finding FDA-approved treatments for rare diseases Winning FDA approval for any type of treatment can be costly and time-consuming, and these challenges are harder to overcome when working on treatments for rare diseases. The type of funding afforded to researchers working on more widely spread conditions is often unavailable to research teams working with rare diseases, reducing the amount of effort, time, equipment, and expertise that might go into research and development. Costs for this type of medical R&D are high. Without funding and with limited patient populations in need of any resulting treatment, the business economics are not in favor of development. Private companies may not choose to expend many resources in these directions because they may see no opportunity for profit. Even when private companies or public institutions commit to researching treatments for rare diseases, the limited patient population can be a barrier to seeking FDA approval. The small number of available patients can make it difficult to run the type of clinical trials required to support an application for FDA approval. By incentivizing rare disease treatment research, the Orphan Drug Act helps to lower some of these seemingly impossible barriers. Diseases with FDA-approved treatments The FDA has approved hundreds of treatments for rare diseases since the Orphan Drug Act was passed. While some treatments may offer a cure, many address conditions for which no known cure exists as of 2024. In these cases, the FDA-approved treatments are meant to address specific symptoms related to the disease process, helping to improve quality of life, potentially extend the expected life span with a disease, or otherwise support more positive outcomes for patients. Lennox-Gastaut Syndrome Lennox-Gastaut Syndrome (LGS) impacts an estimated 48,000 people in the United States. It’s a rare and severe form of epilepsy; less than 2% of people with epilepsy have this condition, but it’s somewhat more prevalent in children. Usually diagnosed at an early age, LGS can lead to developmental delays and lifelong disabilities in some people. While no cure currently exists for LGS, there are some FDA-approved treatments to help control symptoms and support better quality of life. Approved treatments, which are used in conjunction with other methods to help reduce seizures, include: RufinamideCannabidiol Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy caused by genetic mutations in chromosomes. This rare disease primarily impacts males, and data published by the Centers for Disease Control and Prevention indicate that in a population of males age 5 to 9, there is a 1 in 5,000 chance of any one boy having Duchenne or Becker muscular dystrophy (BMD). While no cure exists, numerous FDA-approved drugs for DMD are available to help support a better quality of life and potentially extend the expected life span of someone with this rare disease. Some approved drugs include: CasimersenDeflazacortEteplirsenViltolarsenGolodirsenGivinostat Gaucher Disease Gaucher disease is a genetic disorder that requires both parents to carry it for the child to get it. The disease reduces a specific enzyme that breaks down certain fats in the body, resulting in potential fatty buildup in organs like the liver or spleen. This can lead to issues with organ function and inflammation, causing a variety of symptoms. According to the National Gaucher Foundation, around 1 in 40,000 births can be impacted by this disease. However, around 1 in 10 people are carriers, and the disease is more common among people of certain ethnicities. While no cure exists for Gaucher disease, the FDA has approved numerous drugs that may be prescribed to help with symptoms related to the condition. They include: MiglustatEliglustatVelaglucerase alfaImigluceraseTaliglucerase Future Developments While much ground has been covered in rare disease treatment research since the Orphan Drug Act was passed, there are still many rare diseases with little to no effective treatment options. Examples include, but certainly aren’t limited to, Alkaptonuria, Hutchinson-Gilford Progeria Syndrome, and Fahr’s disease. More work is required in this area, and medical researchers continue to seek cures, palliative treatments, and drug protocols that help with symptoms related to rare diseases. New technologies, including artificial intelligence, are helping to speed up research and may increase the rate at which new drugs are developed for rare diseases.

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