CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism
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CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism

  In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…

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Positive Data Announced from AYVAKIT Clinical Trials for Systemic Mastocytosis
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Positive Data Announced from AYVAKIT Clinical Trials for Systemic Mastocytosis

  In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…

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Kevin Hart Gives a Hand to the Muscular Dystrophy Association As Host of the MDA Telethon
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Kevin Hart Gives a Hand to the Muscular Dystrophy Association As Host of the MDA Telethon

As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…

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EMA Grants PRIME Designation to CTX001, a Sickle Cell Disease Treatment
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EMA Grants PRIME Designation to CTX001, a Sickle Cell Disease Treatment

CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…

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Clinical Remission Versus Imaging Remission in Rheumatoid Arthritis
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Clinical Remission Versus Imaging Remission in Rheumatoid Arthritis

by Lauren Taylor from In The Cloud Copy Rheumatoid arthritis (RA) is an auto-immune condition that occurs when the body’s immune system mistakenly targets the joint linings causing inflammation, pain,…

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Experimental Compound Shows Benefit in Mouse Model of Charcot-Marie-Tooth Disease Type 1A
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Experimental Compound Shows Benefit in Mouse Model of Charcot-Marie-Tooth Disease Type 1A

According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…

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