As humans, we evolve and grow exponentially over time, and viruses and bacteria grow at a thousand times the rate of us, resulting in many medical advancements and the introduction of new diseases and health issues. Who would have predicted that the world would be struck by a massive global pandemic in 2019, causing havoc and the loss of millions of lives worldwide?
Are they a significant threat to one’s health?
Rare diseases, often known as orphan diseases, are classified based on their rarity of occurrence. Although these diseases are rare individually, they are a significant concern in the United States and the European Union, affecting 6-8 percent of the population or around thirty million individuals.While the regulatory definition differs by location, the prevalence is likely to be comparable.
A rare disease is classified in the United States as one that affects fewer than 200,000 individuals, but in the European Union it affects fewer than five subjects per 10,000 people.Orphan illness regulations typically limit such classification to highly disabling or life-threatening diseases that lack an effective therapy or cure.
What is the significance of the issue?
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Orphan illness regulations typically limit such classification to highly disabling or life-threatening diseases that lack an effective therapy or cure. The significance of rare diseases stems from the fact that certain rare diseases are so uncommon that a single physician may never have seen a single patient with that ailment.
Rare disease detections in Hong Kong:
The department of rare diseases treatments hong kong now refers to “rare diseases” as “uncommon diseases,” and there is no official definition of “uncommon diseases.” Although not formally defined, there are many RD cases in Hong Kong, including Mucopolysaccharidoses, Pompeo Disease, Angelman Syndrome, Albinism, Marfan Syndrome, Rett Syndrome, Spinal cerebellar Ataxia, Tuberous Sclerosis Complex, Spinal Muscular Atrophy, Fabry Disease, Gaucher’s Disease, and others.
Pfizer’s feat in solving this issue:
Pfizer rare diseases gene treatments hong kong focuses on smaller patient groups yet aspires to have a large effect. They are attempting to fundamentally alter what it means to live with a rare condition. Develop breakthrough therapies, they combine innovative research with a thorough grasp of the underlying disease pathophysiology.
Pfizer’s extensive global rare illness portfolio, with over three decades of experience in rare disease, strives to address unmet medical needs in a variety of therapeutic areas, including haematology, neurology, endocrinology, cardiology, and inherited metabolic disorders.
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Pfizer is aiming to pioneer gene therapy discoveries that will improve the lives of patients. They hope to unleash the promise of gene therapy for people throughout the world who have rare genetic illnesses and whose current standard of care falls short.
