BeginNGS and Sidra Medicine Unite to Transform Newborn Genetic Screening in Qatar

The Rady Children’s Institute for Genomic Medicine (RCIGM) has announced a landmark international collaboration with Sidra Medicine in Qatar to launch the BeginNGS® newborn genome sequencing program—marking a significant step…

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AlphaDetect: A Bold New Era for Centralized Alpha-1 Antitrypsin Deficiency Detection

The Alpha-1 Foundation (A1F) has announced a groundbreaking advancement in the fight against Alpha-1 Antitrypsin Deficiency (AATD) with the launch of AlphaDetect, a dedicated non-profit subsidiary focused on revolutionizing the…

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Rare Community Profiles: AlphaID At Home Provides Accessible, Targeted Screening for Alpha-1 Antitrypsin Deficiency (AATD)
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Rare Community Profiles: AlphaID At Home Provides Accessible, Targeted Screening for Alpha-1 Antitrypsin Deficiency (AATD)

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

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Experimental Treatment for Alpha-1 Antitrypsin Deficiency Earns Fast Track Designation
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Experimental Treatment for Alpha-1 Antitrypsin Deficiency Earns Fast Track Designation

According to a story from globenewswire.com, the biopharmaceutical company Mereo BioPharma Group plc has recently announced that the company's investigational therapeutic alvelestat has earned Fast Track designation from the US…

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Results From This Study are Encouraging for Alpha-1 Antitrypsin Deficiency Liver Disease
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Results From This Study are Encouraging for Alpha-1 Antitrypsin Deficiency Liver Disease

  Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…

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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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Patients with Alpha-1 Antitrypsin Deficiency Liver Disease Show Improvement After Six Months of Experimental Treatment
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Patients with Alpha-1 Antitrypsin Deficiency Liver Disease Show Improvement After Six Months of Experimental Treatment

  Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…

Continue Reading Patients with Alpha-1 Antitrypsin Deficiency Liver Disease Show Improvement After Six Months of Experimental Treatment