by Priyanka Kabra 29.08.22
Used to treat a disease named Spinal Muscular Atrophy (SMA). SMA occurs due to a mutation on a fifth chromosome (5q) characterized by progressive muscle atrophy that starts shortly after birth.
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Used to treat Pompe disease (rare, inherited disorder). This disease ultimately leads to impaired function of the muscles and liver.
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Used to treat a rare inherited disease that affects only males- Hunter syndrome which causes walking and breathing difficulties.
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Used to treat a rare inherited disease that affects only males- Hunter syndrome which causes walking and breathing difficulties.
Used to treat a rare group of diseases that affect red blood cells.
Used to treat a rare group of diseases that affect red blood cells.
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Used to treat patients with urea cycle disorders (UCDs)
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Used to treat patients with urea cycle disorders (UCDs)
Used to treat Hutchinson-Gilford Progeria Syndrome. HGPS is an extremely rare disorder which results in young people aging quickly, which results in the death of most patients before the age of 15 years from heart diseases.
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It is the most expensive drug in the world. It comes in the form of suspension for intravenous infusion and targets children under the age of 2 with Spinal Muscular Atrophy (SMA).
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