Positive Data Available from Phase 4 Trial for Primary Immunodeficiencies
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Positive Data Available from Phase 4 Trial for Primary Immunodeficiencies

  In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin…

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Improved Genetic Testing Brings a More Accurate Diagnosis of Primary Immunodeficiency Disorders

  Genetic testing has always been costly and directed at DNA sequencing. Only a limited number of genes could be tested. It was useful to a small number of patients…

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Johns Hopkins Researchers Use Primary Immunodeficiencies to Discover New Info About Immune System Cells

  The COVID era emphasized the importance of maintaining a strong immune system. But what about people who are less fortunate? Johns Hopkins researchers recently announced some good news for…

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Study of the Week: Early GI Cancer Screening is Recommended for Primary Immunodeficiency Patients
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Study of the Week: Early GI Cancer Screening is Recommended for Primary Immunodeficiency Patients

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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Primary Immunodeficiency Patients Should be Screened for Gastrointestinal Cancers Early

A research study led by Professor Beishi Zheng from Columbia University has studied the immunogenetics of gastrointestinal (GI) cancers. The Study This study demonstrated that individuals who are diagnosed with…

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Three Poster Presentations Demonstrate The Long-Term Efficacy of Mavorixafor in Primary Immunodeficiencies
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Three Poster Presentations Demonstrate The Long-Term Efficacy of Mavorixafor in Primary Immunodeficiencies

X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…

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First Patient Dosed in IVIG Study for PI
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First Patient Dosed in IVIG Study for PI

  According to a July 13 news release, biopharmaceutical company Kedrion Biopharma ("Kedrion") recently treated its first pediatric patient within the Phase 3 KIDCARES10 clinical trial. Within the trial, Kedrion…

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Last Patient Treated in CARES10 Trial for Primary Immunodeficiency (PI)
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Last Patient Treated in CARES10 Trial for Primary Immunodeficiency (PI)

According to a recent press release, biopharmaceutical company Kedrion Biopharma ("Kedrion") achieved an important milestone in its Phase 3 CARES10 trial: the last treated patient. During the CARES10 trial, researchers evaluated…

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UK Moves to Make Whole Genome Sequencing Standard in the NHS, Improving Diagnosis for Rare Disease Patients

A recent study has demonstrated that through standardizing the whole genome sequencing process and making it routine for patients, we can improve rare disease diagnosis across the board. Earlier diagnosis…

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New Method of Infusion for Primary Immunodeficiency Could Improve Patients; Quality of Life
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New Method of Infusion for Primary Immunodeficiency Could Improve Patients; Quality of Life

A New Study A new study, conducted by CLS Behring, has found that a new method of administration for Hizentra as a treatment for primary immunodeficiency (PI), could improve quality…

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What if the World Always Thought of the Immunocompromised as They are Right Now?
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What if the World Always Thought of the Immunocompromised as They are Right Now?

If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It's the small precautions, like washing your hands That for me…

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ICYMI: Privigen Granted Orphan Drug Designation For Treatment of Systemic Scleroderma
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ICYMI: Privigen Granted Orphan Drug Designation For Treatment of Systemic Scleroderma

CSL Behring has recently received the Orphan Drug designation for Privigen for the treatment of systemic scleroderma. Privigen has also been granted the Fast Track designation. This therapy has been…

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ADMA Biologics Earns BioNJ 2020 Innovator Award for Primary Immunodeficiency Treatment
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ADMA Biologics Earns BioNJ 2020 Innovator Award for Primary Immunodeficiency Treatment

According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…

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Orphan Drug Designation Provided to a Treatment for Chronic Inflammatory Demyelinating Polyneuropathy
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Orphan Drug Designation Provided to a Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to…

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