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TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Abbott Spinal Cord Stimulation (SCS) Systems Manufacturer/developer : Abbott Medical Date of FDA approval : May 30, 2024 Approved for : Chronic, hard-to-manage pain in the torso, arms and legs.
Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.
Both pipeline products use Biotest’s newly developed production facility, which enables improved manufacturing of fibrinogen with higher purity, defined concentrations, viral safety, and good solubility.
The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.
Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and gene expression patterns. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.
Absence of Biomarkers for Diagnosis: Due to the absence of genotype-phenotype correlations and prognostic markers, there have been challenges in the diagnosis and treatment of rare kidney diseases. Insufficient Model Organisms: Limitations of these model organisms includelong generation time and strain effects.
Management options to date were limited to hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). Until today, there were no approved pharmaceutical therapies for PH1.
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