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Investigational drug restores calcium levels in rare genetic disorder

An investigational drug, encaleret, restored calcium levels in people with autosomal dominant hypocalcemia type 1 (ADH1), a rare genetic disorder marked by an imbalance of calcium in the blood and urine, as well as abnormally low levels of parathyroid hormone, which regulates blood calcium levels.

Lily ramseyRena dsouzaRachel gafniMichael collinsIris hartleyDevelopment agreementNational institutes of health clinical centerDivision of intramural bio pharma incCraniofacial researchCalcilytix therapeutics incNational instituteNational institutesClinical centerNew england journalRenad souzaIntramural research

Experimental Drug Revives Parathyroid Function in Rare Illness

Experimental Drug Revives Parathyroid Function in Rare Illness
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Michael collinsRena dsouzaIris hartleyRachel gafniNational institutes of health clinical centerDevelopment agreementCraniofacial researchCalcilytix therapeutics incDivision of intramural bio pharma incNational instituteNational institutesClinical centerNew england journalRenad souzaIntramural researchBridgebio pharma

Investigational drug restores parathyroid function in rare disease

Investigational drug restores parathyroid function in rare disease
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Michael collinsRachel gafniRena dsouzaIris hartleyRacheli gafniSenior research physicianCraniofacial researchNational institutes of health clinical centerResearch physicianNational instituteNational institutesClinical centerNew england journalRenad souzaAutosomal dominant hypocalcemia type

BridgeBio Pharma (BBIO) Announces NEJM Publication of Positive Encaleret Proof-of-Concept Phase 2b Results in Patients with Autosomal Dominant Hypocalcemia Type 1

BridgeBio Pharma (BBIO) Announces NEJM Publication of Positive Encaleret Proof-of-Concept Phase 2b Results in Patients with Autosomal Dominant Hypocalcemia Type 1
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United statesRachel gafniScott adlerCraniofacial researchBridgebio pharma incEndocrine society meetingMineral researchNational institutes of healthPamerican society for boneBridgebio pharmaNew england journalNational institutesNational instituteAmerican society

BridgeBio Pharma Announces New England Journal of Medicine Publication of Positive Encaleret Proof-of-Concept Phase 2b Results in Patients with Autosomal Dominant Hypocalcemia Type 1 (ADH1)

- In our study, encaleret restored physiologic mineral homeostasis in 13 participants with ADH1, specifically correcting hypocalcemia and reducing hypercalciuria - Rapid and sustained impact of.

United statesVikram baliScott adlerRachel gafniPamerican society for boneMineral researchEuropean unionNational institutes of healthSecurities exchangeGeisinger health systemCraniofacial researchBridgebio pharma incDrug administrationEndocrine society meetingBio pharmaNew england journal