• Diseases
  • Featured
  • CME / CE
  • Podcasts
  • Resources
    • Orphan Drug Approvals
    • Priority Review Voucher
    • Rare Disease Recognition and Awareness Days
  • About Us
  • Diseases
  • Featured
  • CME / CE
  • Podcasts
  • Resources
    • Priority Review Voucher Tracking
    • Rare Disease Recognition and Awareness Days
    • 2026 Orphan Drugs: PDUFA Dates and FDA Approvals
  • About Us
  • Sign Up for our Newsletter
  • Spotlight
  • SMA Research Highlights
  • April Is Fabry Awareness Month
Newsletter
Newsletter

Phase 3 Data of Vonvendi for the Treatment of Von Willebrand Disease

by Madaline Spencer | Dec 19, 2025

Shayla Bergmann, MD, Pediatric Hematologist/Oncologist at the Medical University of South Carolina, discusses phase 3 data of Vonvendi for the treatment of Von Willebrand disease (VWD).     VWD is a genetic condition caused by a missing or defective clotting...

Broader Dosing Ranges of Skytrofa Now Available for Growth Hormone Deficiency

by Madaline Spencer | Dec 18, 2025

Lisa Abbott, MD, endocrinologist with Northern Nevada Endocrinology, discusses the expansion of broader dosing ranges of Skytrofa (lonapegsomatropin) now available for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD).  ...

FDA Approves Uplizna for the Treatment of Generalized Myasthenia Gravis

by Madaline Spencer | Dec 17, 2025

The U.S. Food and Drug Administration (FDA) has approved Uplizna (inebilizumab) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. gMG is a...

Sotatercept for Pulmonary Arterial Hypertension Within the First Year After Diagnosis

by Madaline Spencer | Dec 17, 2025

A recent study, published in the New England Journal of Medicine,  looked at the effects of sotatercept for pulmonary arterial hypertension (PAH) within the first year after diagnosis. PAH affects the heart and lungs. It is characterized by abnormally high blood...

FDA Approves Oral Prophylactic Therapy (berotralstat) for Pediatric Patients With Hereditary Angioedema

by Madaline Spencer | Dec 16, 2025

The U.S. Food and Drug Administration (FDA) has approved an oral pellet formulation of Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to less than 12 years of age. HAE is a condition characterized by...
Page 20 of 120« First«...10...1819202122...304050...»Last »
 Categories
  • Autoimmune / Autoinflammatory Disorders
  • Cancers
  • Congenital And Genetic Conditions
  • Endocrine Disorders
  • Gastrointestinal Diseases
  • Heart Diseases
  • Hematologic Disorders
  • Kidney And Urinary Diseases
  • Lung Diseases
  • Lysosomal Storage Disorders
  • Metabolic Disorders
  • Musculoskeletal Diseases
  • Neurology/Nervous System Diseases
  • Ophthalmology/Eye Diseases
  • Skin Conditions
d Topics
  • Advocacy
  • Diagnosis
  • Drug Development
  • Regulations
  • Treatment
 Contact

Advertising / Promoted Programs
[email protected]

CE / Continuing Education
[email protected]
  • Follow
  • Follow
  • Follow
  • Follow
  • Follow
  • Login
Copyright © 2026 CheckRare. All Rights Reserved.
  • Terms and Conditions
  • Privacy
  • Do Not Use My Personal Information
  • Login
  • Sign Up
Forgot Password?
Lost your password? Please enter your username or email address. You will receive a link to create a new password via email.
body::-webkit-scrollbar { width: 7px; } body::-webkit-scrollbar-track { border-radius: 10px; background: #f0f0f0; } body::-webkit-scrollbar-thumb { border-radius: 50px; background: #dfdbdb }
We use cookies on our website to give you the most relevant experience by remembering your preferences and repeat visits. By clicking “Accept”, you consent to the use of ALL the cookies.
Do not sell my personal information.
Cookie Settings Accept
Manage consent

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may affect your browsing experience.
Necessary
Always Enabled
Necessary cookies are absolutely essential for the website to function properly. These cookies ensure basic functionalities and security features of the website, anonymously.
CookieDurationDescription
cookielawinfo-checkbox-analytics11 monthsThis cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Analytics".
cookielawinfo-checkbox-functional11 monthsThe cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Functional".
cookielawinfo-checkbox-necessary11 monthsThis cookie is set by GDPR Cookie Consent plugin. The cookies is used to store the user consent for the cookies in the category "Necessary".
cookielawinfo-checkbox-others11 monthsThis cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Other.
cookielawinfo-checkbox-performance11 monthsThis cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Performance".
viewed_cookie_policy11 monthsThe cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. It does not store any personal data.
Functional
Functional cookies help to perform certain functionalities like sharing the content of the website on social media platforms, collect feedbacks, and other third-party features.
Performance
Performance cookies are used to understand and analyze the key performance indexes of the website which helps in delivering a better user experience for the visitors.
Analytics
Analytical cookies are used to understand how visitors interact with the website. These cookies help provide information on metrics the number of visitors, bounce rate, traffic source, etc.
Advertisement
Advertisement cookies are used to provide visitors with relevant ads and marketing campaigns. These cookies track visitors across websites and collect information to provide customized ads.
Others
Other uncategorized cookies are those that are being analyzed and have not been classified into a category as yet.
Save & Accept