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Alagille Syndrome
Journal Scan
Oral odevixibat well tolerated, effective in patients with cholestatic liver disease
FDA awards 11 grants for rare disease treatment development
Optical coherence tomography useful for evaluating ocular issues in ALGS
Children with GHD have lower self-esteem than healthy peers
Did lockdowns effect planned surgery in patients with solid cancers?
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Growth Hormone Disorders
October 14, 2021

Children with growth hormone deficiency (GHD) have lower self-esteem and internalized problems than healthy children, according to a study. In this study, 46 prepubescent children with GHD took the Goodman's "Strengths and Difficulties...

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Alagille Syndrome
October 19, 2021

Oral odevixibat reduced serum bile acids pediatric and improved pruritus in patients with cholestatic liver disease and pruritus were evaluated, according to the results of a Phase 2 study. In this phase 2,...

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Lupus Nephritis
October 8, 2021

Among the 91 patients with lupus glomerulonephritis who received add-on belimumab in a real-life setting who met the study criteria, 70.3% (n = 64) achieved primary efficacy renal response (PERR), of which 38.4%...

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Sarcoma
October 12, 2021

Have short-term oncological outcomes been impacted and how will delays in surgery change long-term reductions in survival? In this international, prospective, cohort study, 20 006 adult patients with cancer who were eligible for...

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Polls

Poll: Interstitial Lung Disease-related Social Media Use
Sep 22, 2021

Podcasts

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

Quizzes

QUIZ: Interstitial Lung Disease Case Study
Sep 12, 2021

Videos

ASCO – Retroperitoneal Sarcomas: the Benefits of Radiotherapy
May 27, 2021
Thyroid Eye Disease
June 7, 2021

Mycophenolate mofetil (MMF) appears safe and effective as a second-line immunosuppressive agent in patients with thyroid eye disease (TED), according to a study. In this retrospective cohort study, 20 patients with active moderate-severe to sight-threatening...

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Interstitial Lung Disease
October 17, 2021

The U.S. Food and Drug Administration awarded 11 new clinical trial research grants to support the development of medical products for patients with rare diseases, according to a press release. The Orphan Products...

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