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Molecular Diagnostic Tests: The Unsung Hero of Precise Clinical Decision Making

Roots Analysis

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.

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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

PH1 is an ultra-rare genetic disease 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.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective? “A