In the wake of Martin Luther King Jr. Day, those with rare diseases have vocalized some of the dreams that they have for the healthcare industry. With all of the developments over the years, many are hopeful that their dreams are finally going to come true. With new innovations like gene therapy, treatments for many rare diseases may soon be available. Here are five dreams that patients want to see in the upcoming years.
Treatment for Alzheimer’s Disease
Many companies have attempted to create a lasting treatment for Alzheimer’s disease, none of which have been successful. Some companies have made it into the late stages of clinical trials, only to abandon the research altogether. Biogen is a company that has stuck with the research of their aducanumab program, which may be close to gaining approval from the FDA. High doses of this medication have shown to bring positive results in those with Alzheimer’s. It is possible that this drug may soon be widely available.
A Cure for ALS
Amyotrophic lateral sclerosis (ALS) is a rare disease that is usually fatal within a few years of diagnosis. It is estimated that the number of people diagnosed with this disease will grow in the upcoming years, making the need for a cure great. While there are medications on the market that slow the progression of this disease, there is no cure. Exservan, riluzole, and radicava are all approved treatments for ALS, but they do not function as cures and have limited impact. A dream of patients is that a cure will someday be available.
The End of HIV
There have been many advances in the medical world when it comes to HIV. A few decades ago, HIV was an epidemic. Since the beginning of the HIV/AIDS crisis, over 600,000 people in the United States have passed away from the virus. Fortunately there has been research and developments that have made it possible for people to live with HIV. The director of the CDC has even predicted that HIV can be defeated in the next three to seven years. Two and three drug combinations have been a very large development in the treatment of HIV. Along with these advances, there is research currently being conducted that may lead to a cure for HIV, which has been a dream of many for decades.
A Cure for Cancer
Cancer is a disease that comes in many different forms, and it affects millions of people. The various types of cancer make a cure very difficult; it is most likely not something that will happen soon. But there have been many advances in treatment, which is something that gives hope to millions. Cancer-related deaths have dropped in recent years, with the number declining by 2.2%. This drop is in part due to the new drugs that have reached FDA approval. 48 treatments were approved last year alone. Patients hope that these approvals continue and that the number of cancer-related deaths drops even more.
More Affordable Treatments
Many treatments, especially for rare diseases, are very expensive. For example, Zolgensma, which is a treatment for spinal muscular atrophy, is priced at $2.1 million. The price of this drug and many others have been argued about for years. Politicians have debated about the government’s role in this issue for years but have done little to actually address the problem.
While the government has not made any decisions in this issue, some companies have created plans to address the problem. EQRx is something that will make “market-based solutions” to the expensive price of drugs. Hopefully this development and any future advances will help to make prescription drugs more affordable for patients.
While there are many more dreams that exist for those with rare disease, these are five that many hope to see. Hopefully people will not have to wait long to see progress.
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