Gene therapy Lenmeldy uses stem cells from the patient to deliver copy of working gene inside patients, to cure metachromatic leukodystrophy, also known as MLD.
On Monday, the US Food and Drug Administration approved a therapy for the genetic disease metachromatic leukodystrophy .
Around two years of age, when toddlers are bustling with energy, those affected by MLD start losing their ability to walk and talk. The condition advances quickly, and very soon, the child is in a vegetative state with no chance of recovery.The recently approved treatment, Lenmeldy, is a gene therapy. In this, stem cells are drawn from the MLD patient and infected with an otherwise harmless genetically modified virus.
The small number of potential patients that will use the treatment option means that the costs of discovery, rigorous testing, and manufacturing of these products must be recouped from a small set of recipients, thereby escalating costs. Companies that fail to recover their costs can go bankrupt, thereby shrinking the pool of options that other rare diseases might have in the future., a cure for the genetic condition Hemophilia, cost $3.
The drug was administered for free, but the family spent $500,000 to live in Italy and pay for medical care. Kiera is four now and leading a normal life with no symptoms. Now that the drug is approved in the US, families with MLD will be able to avail it.
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