Winson Tang MD is an experienced medical professional who’s had a successful career in clinical research and drug development for inflammation, nephrology, antivirals, and orphan diseases. Dr Tang has held multiple senior positions at various companies in his career, including Amgen Inc., Tularik Inc., Isis Inc., and Sangamo BioSciences Inc., among others. Among his career highlights is developing drugs for orphan diseases such as liposomal lipase deficiency. As a consultant to Amsterdam Molecular Therapeutics in 209, he oversaw the conduct of the Glybera registration trial. Glybera is currently approved for commercialization in the EU and remains the only gene therapy product approved in the western world.

What is an orphan disease?

An orphan disease can be a rare disease that affects a small number of people in a population and any issues related to the disease are because of its rarity. In Europe, rare diseases are those that affect 1 person among 2000. A disease can be rare in a particular region, but more common in another. This is particularly true with a disease like thalassemia, which is anemia with hereditary origins. It is common in the Mediterranean but occurs erratically in Northern Europe.

The number of orphan/rare diseases number in the thousands. Medical literature routinely describes new diseases, and the number of such instances is also dependent on the specifics of the disease. For the longest time, medical professionals have held that a disease is a change to the state of health that presents unique symptoms. How a pattern becomes defined as unique depends on its analysis.

Origins of orphan diseases

Orphan diseases are often serious, and many of them have genetic origins. There are also multi-genetic diseases that belong in this category such as auto-immune disorders. However, researchers have not fully identified the cause of many orphan diseases.

For many orphan diseases, the signs are present in childhood or at birth, like in the case of Rett syndrome or proximal spinal muscular atrophy. However, a significant number of rare diseases also present during dult hood, like Crohn disease, thyroid cancer or Huntington diseases.

Because of the rarity of orphan diseases, there is a dearth of scientific knowledge upon which to help come up with treatment. While there might be no cure, appropriate medical care and treatment can help improve the life of patients affected by orphan diseases.

About: Winson Tang is Board Certified in Internal Medicine by the American Board of Internal Medicine. Winson Tang MD attained his MD from the Albert Einstein College of Medicine.

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