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Further, such tests also aim to identify single nucleotide polymorphisms, deletions, rearrangements, and insertions in genetic sequences. It is worth mentioning that molecular diagnostics is a subset of the broader in vitro diagnostics market. This vast array of products highlights the critical role of diagnostics in healthcare.
PH1 is an ultra-rare geneticdisease characterized by oxalate overproduction. With the approval of Oxlumo, the FDA has granted Alnylam a pediatric rare disease priority review voucher that entitles the Company to designate a single new drug application to qualify for a priority review in the future.
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare geneticdiseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective? “A
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