A new study published in Molecular Genetics and Metabolism, conducted by a Liverpool based research collaboration involving the University of Liverpool, has identified the drug that treats the extremely rare genetic disease alkaptonuria (AKU) which is called nitisinone stops the progress of AKU also known as black bone disease.
AKU is an iconic genetic disorder characterised by deficiency of the enzyme homogentisate dioxygenase, resulting in accumulation of homogentisic acid (HGA).
Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
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Orphan drugs in the United States of America
As early as 1983, the Public Health authorities realised that legislation on orphan drugs was needed signing of the ‘Orphan Drug Act’. This law defines the ‘orphan drug’ with regard to prevalence (frequency) of the disease for which it is indicated in the American population. In the US, the concept of ‘orphan drug’ does not simply cover pharmaceutical or biological products. It also covers medical devices and dietary or diet products. The OOPD (Office of Orphan Products Development) was created within the FDA (Food and Drug Administration). It is in charge of promoting the availability of safe and efficacious products for the treatment of rare diseases. The ‘orphan’ status allows the drug sponsor to benefit from incentives for the development of these products until the marketing approval.
Availability of orphan drugs in Europe
The granting of marketing approval does not mean the drug is available throughout all the European countries (list of marketed orphan drugs in Europe). The marketing approval holder must decide beforehand on its commercialisation status within every country and the drug will then go through numerous steps in each country in order to condition its management, and usually its price.
Background: The circadian rhythm regulation plays a crucial role in people`s healthy lives affected by factors consisting of cosmic events related to the universe and earth, environmental factors (light, night and day duration, seasons) and lifestyles. These factors changes lead to disturbance of circadian rhythm and it causes increasing the incidence of mental diseases like depression and physiological problems like cancers, cardiovascular disease and diabetes.
RADOIR NEWS_ Dr. Hamidreza Edraki, Head of Radiology Department of “15 Khordad” hospital stated: “Today, MRI techniques are applied for many common cases, such as diagnosis of the diseases in fetus during pregnancy.
Using MRI during the 1st three months of pregnancy is prohibited, indeed as it can affect embryonic germ cells. Thus, it will be harmless to use MRI, afterwards. Congenital disorders are easily diagnosed by MRI and a team including radiologist, Neurosurgeon, gynecologist will control and manage it during the pregnancy and later on.
Orphan Drugs in Third world countries:
Millions of people in Third World countries do not have access to the health care, drugs and vaccines available in developed countries. 10 million children less than 5 years old die each year of infectious diseases for which treatments exist in developing countries. 3 million children die each year because they have not been immunised. 150 million women would like to wait longer between two pregnancies or limit them, but they do not have access contraception. The spread of the AIDS epidemic only widens the health gap between poor countries and developed countries.
Insights into Rare Disease Drug Approval:Trends and Recent Developments Mike Lanthier Operations Research Analyst FDA Office of the Commissioner NORD Rare Diseases & Orphan Products Breakthrough Summit, October 17, 2017
The so-called ‘orphan drugs’ are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions.
The process from the discovery of a new molecule to its marketing is long (10 years in average), expensive (several tens of millions of euros) and very uncertain (among ten molecules tested, only one may have a therapeutic effect).
The 2nd award of Corporate Social Responsibility (CSR) was held on 24th July, 2018 with the presence of major domestic companies .
RADOIR News- Corporate Social Responsibility (CSR) is a self-regulating business model that helps a company be socially accountable to itself, its stakeholders, and the public.