Patient Story: Liver Transplant Allows Girl Diagnosed with Biliary Atresia to Celebrate her First Birthday
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Patient Story: Liver Transplant Allows Girl Diagnosed with Biliary Atresia to Celebrate her First Birthday

According to a recent article, a child with biliary atresia was able to celebrate her first birthday following her liver transplant. Biliary Atresia Biliary atresia is a condition occurring in…

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Study Highlights Experiences of Patients Treated with ADV7103 for Distal Renal Tubular Acidosis
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Study Highlights Experiences of Patients Treated with ADV7103 for Distal Renal Tubular Acidosis

According to a recent article, a new study is demonstrating how the effects of distal renal tubular acidosis impact a patient’s quality of life. Distal Renal Tubular Acidosis (dRTA) Distal…

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Phase 3 Investigation of Ampreloxetine Produces Positive Results for Neurogenic Orthostatic Hypotension

Theravance Biopharma Inc. has just released results from their 2nd Phase 3 investigation of Ampreloxetine as a therapy for symptomatic neurogenic orthostatic hypotension (nOH). Symptomatic nOH NOH is a rare…

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Advocates Ask that the Same Effort Used for COVID Drug Discovery Be Used for Rare Diseases

Dr. Christopher Austin, a National Institute of Health Director for Transitional Sciences, spoke at last year’s virtual conference honoring Rare Disease Day. According to an article in the NIH Record, Dr.…

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