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news.Spyryx gets development award to advance novel therapy for cystic fibrosis
In the CF lung, the epithelial sodium channels (ENaC) hyperabsorb sodium and water from the surface of the lung airway, this leads to dehydration of the mucus layers
In the CF lung, the epithelial sodium channels (ENaC) hyperabsorb sodium and water from the surface of the lung airway, this leads to dehydration of the mucus layers
QR-010, a RNA-based oligonucleotide, is designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene
The MAA is based on results from two global Phase III clinical trials that examined the efficacy, safety and pharmacokinetics of Alprolix for hemophilia B. The two trials
The proposed indication for Strensiq is for long-term enzyme replacement therapy in patients with pediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease. anuma is proposed
RVO is a significant cause of vision impairment and a chronic disease that requires early and ongoing management to obtain the best possible vision. The company said that
Humira is designed for the treatment of these adults with an inadequate response to conventional systemic HS therapy. HS, referred to as acne inversa, is a painful, chronic
Farydak is the first histone deacetylase (HDAC) inhibitor approved in combination with bortezomib, a type of chemotherapy, and dexamethasone, an anti-inflammatory medication, to treat these patients who received
In this sturdy, NIAAA is working in partnership with XenoPort, a biopharmaceutical company, which will supply gabapentin enacarbil. Around 346 patients will be enrolled in this randomized, double
The Italian biopharmaceutical firm has also filed an application for orphan designation in the US. RTT primarily affects females with an estimated prevalence ranging from 1:10-20,000 females and
The company said that the study initiation is the training of hospital staff to allow for patient screening and immediate patient enrollment into the clinical trial upon selection.