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.

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Ophthalmology Month in Review: September 2024 | Image Credit: HCPLive
Ophthalmology Month in Review: September 2024

October 8th 2024

This September 2024 month in review highlights recent advances in gene therapies and therapies demonstrating the potential for improved vision outcomes.

TransCon hGH Aims for FDA Label Expansion for Adult Growth Hormone Deficiency| Image Credit: US Food and Drug Administration
TransCon hGH Aims for FDA Label Expansion for Adult Growth Hormone Deficiency

September 30th 2024

Retinitis Pigmentosa Diagnosis Intensifies Risk of Depressive Disorder| Image Credit: The American Society of Retina Specialists
Retinitis Pigmentosa Diagnosis Intensifies Risk of Depressive Disorder

September 29th 2024

FDA Approves Levacetylleucine for Niemann-Pick Disease Type C | Image Credit: US Food and Drug Administration
FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

September 25th 2024

FDA Approves Arimoclomol, First Drug for Niemann-Pick Disease, Type C | Image Credit: US Food and Drug Administration
FDA Approves Arimoclomol, First Drug for Niemann-Pick Disease, Type C

September 20th 2024

Video Interviews
Podcasts
New Insight with Veeral Sheth, MD Podcast logo | Image Credit: HCPLive
Autoimmune Hepatitis at ACG 2023, with Gina Choi, MD

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