In this list, we mention the top 5 rare disease-based articles that are a must-read for anyone interested in the life science field. Find out in detail about these diseases, what causes them, and how they are treated presently.
Hemophilia: The Evolved Gene Therapy for Hemophilia
Hemophilia is a rare inherited disease in which blood clotting occurs irregularly. The person affected fails to make clotting factors (blood-clotting proteins), thus continuing to bleed whenever sustained by an injury.
Historically, there has been no cure for Hemophilia. The only treatment called Replacement Therapy involves periodically replacing the missing blood clotting factor, thus ensuring the proper clotting of blood. The treatment can either be prophylactic or episodic. To achieve this clotting factors are injected into a person’s veins.
In theory, Gene therapy can restore normal blood clotting processes, making it a revolutionary and advanced treatment option. However, there are a few drawbacks along with benefits. Could it then be the better treatment option?
Article: Gene Therapy for Hemophilia – Benefit, Challenges, Risk, and Advances.
Phenylketonuria: Which Pipeline Therapy is Expected to Capture the Eyeballs in the Phenylketonuria Market?
Phenylketonuria is the deficiency or absence of phenylalanine hydroxylase (PAH), an enzyme responsible for amino acid phenylalanine processing. It is a rare inherited condition that affects one in 19,000 newborns in the US, as per NORD.
Even though rare, a lack of proper treatment can cause severe intellectual disability, thus adding a tremendous burden to healthcare. To prevent any intellectual disability, the newborn is treated with a phenylalanine-restricted diet under a carefully controlled environment during the first few weeks of life. For adults with Phenylketonuria, Lofenalac (a dietary supplement) is added to the diet to absorb phenylalanine to a certain extent.
However, the scientific community is now exploring additional approaches to treat Phenylketonuria.
Article: Phenylketonuria (PKU) Market – Key Companies in Phenylketonuria Market.
Myasthenia Gravis: Exploring the Market Potential of the Upcoming Therapies for the Myasthenia Gravis
Myasthenia Gravis, an autoimmune disease, causes fatigue and muscle weakness by disrupting the communication between muscles and nerve cells. As a result, critical muscle contractions do not properly occur, thus causing muscle weakness. There is still a lack of understanding of the role that the thymus gland plays in the progression of Myasthenia Gravis. Also, there is an unmet need of developing novel biomarkers that could ease diagnosis.
The Myasthenia treatment market consists of several therapies that address the burden caused by the disease. Many companies are striving to develop drugs that can effectively help patients.
The present treatment for Myasthenia Gravis pivots on immune system suppression and regulation on a broad scale. However, future treatment approaches may focus on targeted immunotherapies that try to improve results and reduce adverse effects.
Article: Myasthenia Gravis Treatment Market – Companies and Therapies Analysis.
von Willebrand disease (vWD): Misdiagnosis Still Prevalent!
von Willebrand disease (vWD) is an inherited bleeding disorder that affects around 3.2 million of the US population. The disease is mired in inaccurate diagnosis and therefore has many diagnostic limitations. This causes vWD to often be misdiagnosed/underdiagnosed for other forms of Hemophilia. The diagnostic limitations result in substantial patient morbidity leading to an easily avoidable burden being added to the healthcare system
So what is von Willebrand disease, why does it end up being misdiagnosed, and what are the current and possible therapies? This article explains.
Article: von Willebrand Disease (vWD) Market – vWD Therapy and Key Companies.
Top Drugs Of 2021: An in-depth Assessment of the Top Drugs Launched by Leading Global Companies in the First Half of 2021 (H1)
Innovation is the leading factor for driving progress. Innovative medications get approved every year to treat new and existing disorders that are an obstacle to humanity. After the 2020’s global pandemic, however, the expectation from leading pharmaceutical companies is especially strong for 2021.
This year, in addition to the pharmaceutical industry’s exceptional response to COVID-19, pharmaceutical developers have also effectively developed advanced medicines to overcome conditions that impact millions worldwide. With that said, these are the most anticipated drug launches in the first half of 2021.
Article: Top Drugs Launched in 2021 (H1) – List of FDA Approved New Therapies.
Many common and rare diseases affect humanity regularly. The diseases discussed above are some of the many rare diseases with exciting emerging treatments. However, many more complex rare diseases grapple people around the world. What are these? What causes them? And is there any cure around the corner?
Find out here:Rare Diseases Blogs, By Delveinsight.
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