Rare Disease

Latest News

LEO Pharma to Acquire Timber Pharmaceuticals
LEO Pharma to Acquire Timber Pharmaceuticals

August 21st 2023

Timber Pharmaceuticals has been developing TMB-001 for the treatment of congenital ichthyosis.

Abeona Files Briefing Package For EB-101 Pre-Biologics License Application
Abeona Files Briefing Package For EB-101 Pre-Biologics License Application

July 31st 2023

Influencing and Improving Photoprotection Behaviors in Patients With Xeroderma Pigmentosum
Influencing and Improving Photoprotection Behaviors in Patients With Xeroderma Pigmentosum

July 12th 2023

Pediatric Necrobiosis Lipoidica Yields Treatment Failure Rate of 40%
Pediatric Necrobiosis Lipoidica Yields Treatment Failure Rate of 40%

July 11th 2023

Alan Mendelsohn, MD, Reviews Phase 3 TMB-001 PK Data for Congenital Ichthyosis
Alan Mendelsohn, MD, Reviews Phase 3 TMB-001 PK Data for Congenital Ichthyosis

July 5th 2023

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Community Practice Connections™: Chronic Fibrosing ILDs With Progressive Phenotype–Are You Aware of the Latest Data on Diagnosis and Emerging Treatments?

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Free COPE CE at AAOPT

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Medical Crossfire®: Diagnostic and Clinical Considerations in Cutaneous T-Cell Lymphoma for the Dermatologist and the Collaborative Cancer Team

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Taking Pompe Disease Management to the Next Level: Optimizing Clinical Assessments and Treatment Decision-Making

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Expert Illustrations and Commentaries™: A Deeper Look at the Pathogenesis of Hidradenitis Suppurativa (HS)

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C.R.U. Eye Symposium (Current, Relevant, Useful)

11/10/2023-11/12/2023

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Best Practice in the Multidisciplinary Care of the Patient with HS

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Addressing Challenges in the Management of HS in Special Populations

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Addressing Healthcare Inequities in Cardiac Amyloidosis: Interventions Aimed at Improving Care for All Patients

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Clinical Consultations™: Raising Awareness and Optimizing Treatment in Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

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Addressing Healthcare Inequities: Overcoming Challenges in the Equitable Management of Idiopathic Pulmonary Fibrosis

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Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices

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Cases and Conversations™: Enhancing Diagnosis and Evaluation of Generalized Pustular Psoriasis in Advanced Practice Settings

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Community Practice Connections: Optimizing Recognition and Management of Acute Hepatic Porphyria

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Addressing Healthcare Inequities: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis

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Advances In™ Desmoid Tumors: Recognizing Disease Burden, Pathophysiology, and Targeted Treatments in Development

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Transfusion-Related Iron Overload–Evidence-Based Approaches to Improve Chelation Outcomes

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Patient and Provider Connection™: Aligning Evidence-Based Management Strategies with Quality-of-Life Considerations to Optimize Care in Patients with HAE

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"D" is for Diagnosis: Deciphering Uncommon Hematologic Disorders Through the Eyes of a Detective

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Community Practice Connections™: Transforming How We Treat Sickle Cell Disease: Holistic Approaches to Addressing Clinical Challenges Across the Patient’s Lifespan

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Addressing Healthcare Inequities™: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis

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Clinical ShowCase™ in Atopic Dermatitis: Personalized Management Plans for Pediatric Patients

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Community Practice Connections™: Multidisciplinary Management of BPDCN: Strategies for Differential Diagnosis and Integrating Novel Targeted Therapy

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20th Annual International Symposium on Melanoma and Other Cutaneous Malignancies®

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Advances in™ Congenital Adrenal Hyperplasia: Investigating the Impact of Emerging Agents

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Facing the Changing Landscape for Neuromodulators and Dermal Fillers

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Expert Illustrations & Commentaries™: Targeting the Complement System to Optimize Treatment of Cold Agglutinin Disease

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BURST CME™: Taking ALS Management to the Next Level

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BURST Expert Illustrations & Commentaries™: A Closer Look at the Role of Complement Inhibitors in the Treatment of Paroxysmal Nocturnal Hemoglobinuria

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Advances In: Integrating New Treatment Options into Management Plans for Patients with Moderate-to-Severe Atopic Dermatitis

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