A Resilient Spirit: The Life of a Patient and Advocate
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A Resilient Spirit: The Life of a Patient and Advocate

Jean's life story is one of extraordinary resilience, a testament to the strength of the human spirit in the face of rare and challenging medical conditions. Born in 1943 in…

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Investigational Therapy Earns Orphan Drug Designation for Hereditary Angioedema
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Investigational Therapy Earns Orphan Drug Designation for Hereditary Angioedema

According to a story from Business Wire, the biopharmaceutical company Astria Therapeutics, Inc., recently announced that its experimental therapy navenibart has earned the US Food and Drug Administration's Orphan Drug…

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Study: Tirzepatide Lowers Risk of Pancreatitis and Diabetic Retinopathy vs GLP-1s
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Study: Tirzepatide Lowers Risk of Pancreatitis and Diabetic Retinopathy vs GLP-1s

According to a story from Healio, analysis of the FDA Adverse Events Reporting System database indicates that tirzepatide has a superior safety profile when compared to GLP-1 inhibitors that have…

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NDA for NF1-PN Treatment Charges Ahead with Priority Review
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NDA for NF1-PN Treatment Charges Ahead with Priority Review

According to a press release from GlobeNewswire, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has granted the company's New Drug Application (NDA)…

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September is Charcot-Marie-Tooth Disease Awareness Month: Spreading Rare Disease Awareness
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September is Charcot-Marie-Tooth Disease Awareness Month: Spreading Rare Disease Awareness

September is recognized as Charcot-Marie-Tooth disease (CMT) Awareness Month. Although this illness is one of the most widespread inherited genetic disorders, there is still a significant need to spread awareness…

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Proof-of-Concept Trial Suggests That Tezepelumab (TEZSPIRE) Could Play a Role in Treating COPD
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Proof-of-Concept Trial Suggests That Tezepelumab (TEZSPIRE) Could Play a Role in Treating COPD

The COURSE Phase IIa clinical trial was conducted to evaluate tezepelumab (marketed as TEZSPIRE) in people living with chronic obstructive pulmonary disease (COPD) that had a wide range of blood…

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August is Gastroparesis Awareness Month!
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August is Gastroparesis Awareness Month!

First established by the International Foundation for Gastrointestinal Disorders (IFFGD) in 2016, Gastroparesis Awareness Month is designed to increase awareness of gastroparesis and how it affects the people who have…

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These Nonprofits Just Shelled Out $5.2 Million to Fund An Experimental Parkinson’s Drug
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These Nonprofits Just Shelled Out $5.2 Million to Fund An Experimental Parkinson’s Drug

According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company's investigational Parkinson's disease therapy. Intended to…

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