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Growth Hormone Disorders
Algorithm helps detect undiagnosed adult growth hormone deficiency
Some patients with TED need longer to respond to teprotumumab
Clinically meaningful ORR to zetomipzomib seen in patients with lupus nephritis
Ripretinib appears safe, effective in KIT-altered metastatic melanoma
Disease course varies greatly in patients with advanced pulmonary sarcoidosis
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Alagille Syndrome
June 23, 2022

Patients with Alagille syndrome (ALGS) treated with the ileal bile acid transporter inhibitor (IBATi), maralixibat, experienced catch-up height and weight, with the greatest catch-up in height observed in those with the greatest height...

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Polls & Quizzes

Quiz: Decentralized Clinical Trials for Rare Diseases
Jun 24, 2022

Podcasts

Rare Disease Drug Shortage During COVID-19 Effort Creates Dilemma
Feb 15, 2021

Quizzes

Quiz: Decentralized Clinical Trials for Rare Diseases
Jun 24, 2022

Videos

Challenges in Developing New Therapeutics for Epithelioid Sarcoma
Jun 24, 2022
Ophthalmology
April 29, 2022

Direct-to-consumer advertising may have helped to increase public interest for thyroid eye disease (TED), according to researchers who noted an increase in internet search traffic for TED after 2 advertising campaigns for teprotumumab....

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Thyroid Eye Disease
June 30, 2022

Patients with thyroid eye disease (TED) treated with teprotumumab may need longer than 12 weeks to see a clinically significant response, according to a study. Approximately half of the patients treated with teprotumumab...

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Growth Hormone Disorders
July 1, 2022

Researchers have developed an algorithm that has the potential to improve the detection of adult growth hormone deficiency (AGHD), an often underdiagnosed disease associated with increased morbidity and mortality, according to a study....

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Interstitial Lung Disease
June 26, 2022

A widely varying disease course was seen in patients with advanced pulmonary sarcoidosis, with patients with progressive fibrosing interstitial lung diseases (PF-ILD) having a higher mortality rate than the overall pulmonary sarcoidosis rate,...

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