Clinical Insights

Second Indication for Aqneursa in Ataxia Telangiectasia

Second Indication for Aqneursa in Ataxia Telangiectasia

The US Food and Drug Administration (FDA) has approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in patients with ataxia-telangiectasia who weigh at least 33 pounds (15 kg).  It is the first treatment approved for this rare condition. Ataxia...

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First-Line Therapy Approved for Rare Lung Cancer | The CheckRare Brief Ep 7

  This week, three developments highlight the rapidly changing rare disease treatment landscape: a Phase 3 setback in Angelman syndrome, continued competition in hereditary angioedema, and an...

New Huntington’s Disease Gene Therapy Application | The CheckRare Brief Ep 6

  In this episode of The CheckRare Brief, we discuss two recent FDA approvals for rare diseases, including Zanvastro for Alexander disease and Besremi for essential thrombocythemia (ET). We...

Data on Maribavir in Post-Transplant Cytomegalovirus

Neil Inhaber, MD, FRCPC, Therapeutic Area Head of Global Medical, Rare Disease at Takeda, discusses data on maribavir in post-transplant cytomegalovirus (CMV) presented at the American Transplant...

New FDA Approvals for Rare Diseases: From Autoimmune Disorders to Pancreatic Cancer | The CheckRare Brief Ep 5

  In this episode of The CheckRare Brief, we discuss four recent FDA approvals, including LISRAYA for dermatomyositis and Imaavy for warm autoimmune hemolytic anemia (wAIHA), Mimrylo for...

Primary IGF-1 Deficiency: Causes, Early Detection, and Treatment

Primary IGF-1 Deficiency: Causes, Early Detection, and Treatment

Robert Rapaport, MD, discusses the differences between severe, primary IGF-1 deficiency and growth hormone (GH) deficiency.

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Fabry Disease: Is The Risk of Stroke or Heart Failure Being Properly Managed By Your Medical Team?

Jenny McCue, Vice President of Global Research and Clinical Development at the Myasthenia Gravis Foundation of America (MGFA), discusses research highlights to be presented at the upcoming 2026 MGFA Scientific Session being held September 29th in Orlando, FL.

🌍✨ Happy World Narcolepsy Day! ✨🌍

Swipe through to learn about narcolepsy, a rare neurological disorder that affects sleep regulation.

Let’s raise awareness and support those living with narcolepsy!

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...#WorldNarcolepsyDay #NarcolepsyAwareness #CheckRare

Neelam Goyal, MD, and Christyn Edmundson, MD, use case studies involving three different patients suspected of having MuSK Ab+, LRP4 Ab+, and Triple Seronegative Myasthenia Gravis to discuss how they assess those patients, ensure proper diagnosis, and decide which treatment option is most ...appropriate for the individual patient.

View the full CME activity and earn credit at https://checkrare.com/learning/p-beyond-achr-recognizing-and-treating-musk-ab-lrp4-ab-and-triple-seronegative-myasthenia-gravis/

Staci Kallish, DO, and Dawn Laney, MS, CGS, CCRC, examine common concerns in patients with Fabry disease.

View the full program at https://checkrare.com/fabry-disease-patients-real-world-problems-and-how-to-manage-them/

Ratna Bhavaraju-Sanka, MD, and Chilvana Patel, MD, summarize clinically relevant findings and discusses their potential implications for enhancing the care of patients with myasthenia gravis.

View the full CME activity and earn credit at ...https://checkrare.com/learning/p-myasthenia-gravis-clinical-research-highlights-2026/