Amicus Therapeutics: On the Frontline for Treating Rare and Orphan Diseases

Amicus Therapeutics is an American biopharmaceutical company, based in Cranbury, New Jersey, which develops advanced therapies to treat several of the rare and orphan diseases. These disorders have been neglected by doctors due to their rare occurrence and affect only a small number of individuals.

 

Two of the diseases that Amicus Therapeutics focuses on, Fabry disease and Pompe disease, are characterized as Lysosomal Storage Disorders. These disorders are caused by defects in lysosomal function due to an enzyme deficiency, which results in an abnormal build-up of various toxic materials in the body’s cells. Amicus Therapeutics is developing novel enzyme replacement therapy (ERT) products for these Lysosomal Storage Disorders (https://yourbeautycraze.com/amicus-therapeutics-introduces-galafold-fabry-disease-patients/).

 

Their leading product, migalastat, treats individuals with Fabry disease and is in the late-stage development phase. Migalastat is an oral pharmacological medication and has been approved for use in the European Union under the brand name Galafold™. Adult and adolescent patients over the age of 16, with Fabry disease, with an amenable mutation of the enzyme alpha-galactosidase A, are eligible for this therapy. Amicus Therapeutics is also investigating the treatment of patients with Fabry disease who do not have an amenable mutation. Under development is a combination of intravenous migalastat co-formulated with a new enzyme replacement therapy for these individuals.

 

In order to help battle Pompe disease, Amicus Therapeutics is using Chaperone-Advanced Replacement Therapy (CHART™) to develop a new treatment therapy called ATB200/AT2221. This treatment includes using a newly engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme, which is combined with an optimized carbohydrate structure called ATB200 and a small molecule pharmacological chaperone called AT2221. In 2013, this cutting-edge treatment completed the Phase 2 safety study of its development process.

 

As well, Amicus Therapeutics is currently in Phase 3 of their study for the product SD-101. When completed, they will be the first company to market a topical therapy for the rare connective tissue disorder Epidermolysis Bullosa (Glassdoor).

 

Amicus Therapeutics is on the frontline for developing cutting-edge therapies to help fight rare and orphan diseases. They are constantly striving to come up with innovative ideas to develop new therapies and help individuals living with these disorders.

 

Contribution of Mikhail Blagosklonny in field of Oncology research

Mikhail Blagosklonny is the modern scientist who concentrated his career in studying age and cancer. Additionally, he is oncology expert at the Institute of Roswell. He took part in the work of an expert in the Roswell Institute from the year 2009. He graduated from First Pavlov State Medical University where he pursued MD in Internal Medicine.Mikhail Blagosklonny further earned a Ph.D. in Experimental Medicine and Medical College. According to his vast skills in the field of medicine, he became the associate professor of medicine at New York Medical College. He then decided to work as the superior scientist at Ordway Research Institute. His experience in the various areas of research contributed much to him becoming an excellent expert in sphere of research.

Mikhail hard work has earned him a post of becoming the Cell Death and Differentiation editorial board. He has made his theory that relates to the TOR indicating the potential relationship between cancer and the age and suggested treatment by the use of Rapamycin. As a sincere researcher and expert in the field of cancer, he has become the ardent supporter of Rapamycin in the use of cancer treatment. The main reason that has put his research among the best is the different potential that ranges from the cellular and molecular biology.Mikhail as a scientist has marked a real ground in the field of research with his interest in Oncology. He is optimistic that the control of cancer and aging is something that is possible. This has pushed him to put in the records of writing about the aging hyper function theory. Furthermore, he analyses the cell cyclotherapy deeply in his explanation. Furthermore, he tries to put forward an explanation about how cancer can be treated. To him, the approach of cancer treatment is easy as it is indicated in the procedures that show the use of the Rapamycin. In his books and articles, he discusses that the possibility of cancers treatment as opposed to the perception of many.

The ardent love for the research and his determination have put him at the best point of being the best-known researcher in the universe. His ambition as a humanitarian is to create a disease free world that is defined by hopefulness in the community. He is also the editor for PLOS ONE; his hard work has placed his achievement at the require niche in the Oncology research.His contribution to cancer and aging has pushed for the in-depth research and how to utilize the use of the Rapamycin in the cancer treatment. His extended study of the signal transduction pathways in the cancer is to target to slow down the disease that related to the aging. This will create a concept that will become a unique in the cancer biology and on the matters that are associated with the therapy. Furthermore, his research will facilitate the new anti-cancer strategies for the best methods of treating the disease and protecting the healthy cells from the effects of the chemotherapy.

Lifeline Screening – Offering Health Care Screening Packages Across the United States and United Kingdom

It is a well-known fact that people must go for a health checkup and screen on a regular basis to ensure that if there is anything wrong with the body, it can be taken care of in time before its too late. Many of the deaths that occur these days are due to chronic illness or other issues that could have been treated if the symptoms were known earlier or if the preventive health checkup was done earlier to reveal the deteriorating health conditions. It is natural that death cannot be avoided, but as medical science has advanced drastically in the last few decades, there are many modern medical tools and treatments available that can extend the lifespan of the people considerably.

However, to make use of the advanced medical treatments, one has to know if there is anything wrong with the health in time to start the treatment early before it becomes irreversible. It is where the importance of preventive medical check ups and screening comes into the picture. One of the leaders in the United States in preventive health care check-ups is Life Line Screening, which has so far provided health screening of over 8 million people in the United States. Life Line Screening has a pool of preventive health care specialists and doctors who can guide you as per your health conditions and symptoms as to which medical screening packages you should go for. Moreover, patients can have their medical consultancy done from their doctors and come only for health care screening as well.

Life Line Screening provides affordable, advanced, economical and reliable health care screenings that are done using the latest medical technology. Some of the health screening services offered by Life Line Screening are heart disease screening, atrial fibrillation screening, lung cancer screening, c-reactive protein screening, elevated live enzymes screening, high blood pressure, type 2 diabetes, health risk assessment, carotid artery disease screening, and more. Life Line Screening is based in Austin, Texas, and is well-known across the country for its health screening services since its inception in 1993. The services offered by Life Line Screening is not only limited to the United States but is available in the United Kingdom.

Learn More: www.crunchbase.com/organization/life-line-screening#/entity