Patient Stories: A1AD Patients Reunite with Transplant Surgeon
Advancements in transplant surgery has given cystic fibrosis patients new hope for successful lung transplants.

Patient Stories: A1AD Patients Reunite with Transplant Surgeon

Many father-son duos are close, but this pairing - reported on in an article from CBS58 - shares a special bond that was strengthen by the shared experience of a…

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Phase 1 Trial Produces Positive Results for Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

Discerna Pharmaceuticals has just announced the results from a Phase 1 trial investigating their GalXC™ RNAi therapy (Belcesiran) for alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The results demonstrated both…

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Promising Data from Phase 2 VX-864 Study for A1AD, Despite Discontinuation
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Promising Data from Phase 2 VX-864 Study for A1AD, Despite Discontinuation

Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…

Continue Reading Promising Data from Phase 2 VX-864 Study for A1AD, Despite Discontinuation