The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
October 25th 2024
On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.
Expert Illustrations & Commentaries™: Exploring the Role of Novel Agents for the Management of IgA Nephropathy
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‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease
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Clinical ShowCase™: Forming a Personalized Treatment Plan for a Patient With ANCA-Associated Vasculitis
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Mohamad Mohty, MD, PhD, Discusses Cytotoxic T Lymphocytes as Potential EBVPTLD Therapy
Mohamad Mohty, MD, PhD, discusses the investigational therapy cytotoxic T lymphocytes (CTLs) therapy for the treatment of Epstein-Barr virus associated post-transplant lymphoproliferative disorder (EBVPTLD).
Nizar Bahlis, MD, Recaps Follow-Up Data on Combo Therapy for R/R Multiple Myeloma
Nizar Bahlis, MD, highlights the phase 3 POLLUX trial evaluating daratumumab plus lenalidomide and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma.
Michael Jordan, MD, Differentiates Emapalumab Treatment in Pediatric Patients Versus Adults with HLH
The pediatrician discusses key differences between treating pediatric and adult patients with hemophagocytic lymphohistiocytosis (HLH) with the recently FDA-approved emapalumab.
CLL Therapy, Venetoclax/Rituximab, Shows Reduced Risk of Disease Progression
Full results from the phase 3 MURANO trial showed a reduced risk in disease progression and 87.9% overall survival in relapsed/refractory chronic lymphocytic leukemia patients treated with venetoclax/rituximab.
Anemia Patients with MDS Benefit from Luspatercept in Transfusion Burden
December 3rd 2018Currently available anemia therapies only provide benefit for about half of patients with a lower-risk myelodysplastic syndromes (MDS)-related condition—and about one-quarter of such patients dependent on red blood cell transfusions.