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2025年11月18日 (火) 13:13時点における
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Orphan designation does not mean the medication is on the market or authorised for use. All medicines, including designated orphan medicines, should be authorised earlier than they can be marketed and made obtainable to patients in the EU. Through the medication's growth, docs could possibly enrol patients in clinical trials investigating the medicine. Patients with glycogen storage illness type Ia lack an enzyme referred to as glucose-6-phosphatase-? The medicine contains a molecule referred to as messenger RNA (mRNA) which has instructions for making the enzyme. The mRNA is contained in tiny particles of fats (lipids) that prevent it from being damaged down too rapidly. When a affected person is given the medication, a few of their cells will learn the mRNA instructions and temporarily produce the enzyme. This is predicted to normalise the storage and administration of sugars in the body and reduce the signs of the condition. The mRNA from the medication doesn't stay within the physique but is broken down shortly after the medicine is given. The results of the medication had been evaluated in experimental models. No clinical trials with the medication in patients with glycogen storage illness kind Ia had been started. More info on how potential new medicines are tested during their development is accessible on Authorisation of medicines. Medicines meant for uncommon diseases will be granted an orphan designation throughout their improvement. EMA's Committee for Orphan Medicinal Products (COMP) is liable for issuing opinions on applications for orphan designations. The Agency sends the COMP opinion to the European Commission, which is accountable for granting the orphan designation. The complete record of orphan designations is available locally register of orphan medicinal products for human use.<br>
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