Latest Summaries

Aug 4, 2021| Cancers, Hematologic Disorders, Treatment

Current Treatment Options for Bone and Soft Tissue Sarcomas

Roman Groisberg, MD, Medical Oncologist and Director of…
Aug 3, 2021| Advocacy, Neurology/Nervous System Diseases

How the Lennox-Gastaut Syndrome Foundation Helps LGS Patients and Families

Tracy Dixon-Salazar, PhD, Executive Director of the Lennox-Gastaut…
Jul 30, 2021| Cancers, Hematologic Disorders

What Doctors Should Know About Multiple Myeloma

Daniel Auclair, MD, Chief Scientific Officer of the…
Jul 28, 2021| Autoimmune / Autoinflammatory Disorders, Cancers, Hematologic Disorders, Skin Conditions, Treatment

Avapritinib: Mechanism of Action

Daniel DeAngelo, MD, PhD, Chief, Division of Leukemia,…
Jul 27, 2021| Cancers, Drug Development, Treatment

Long-Term Efficacy and Safety of Brentuximab Vedotin in Hodgkin Lymphoma Patients

Thanos Zomas, MD, from Takeda Oncology, discusses follow-up…
Jul 23, 2021| Advocacy, Cancers, Hematologic Disorders

How the MMRF is Advancing Multiple Myeloma Research

Daniel Auclair, MD, Chief Scientific Officer of the…
Jul 22, 2021| Cancers, Diagnosis

Making a Sarcoma Diagnosis

Roman Grosiberg, MD, Medical Oncologist and Director of…
Jul 21, 2021| Cancers

GLOW Trial Demonstrates Favourable Outcome for Elderly CLL Patients Compared to Chemotherapy

Arnon Kater, MD, PhD, Professor of Internal Medicine…
Jul 21, 2021| News, Regulations

Refer4Rare Database Aims to Accelerate Rare Disease Research

Tarun Kumar Bansal, President at Sagacious IP, discusses…
Jul 20, 2021| Congenital And Genetic Conditions, Metabolic Disorders

FDA Approves Odevixibat, First Drug Therapy for PFIC

The Food and Drug Administration (FDA) has approved…
Jul 20, 2021| Cancers, Hematologic Disorders

Location, Location, Location: Your Multiple Myeloma Survival Depends on It

Daniel Auclair, MD, Chief Scientific Officer of the…
Jul 19, 2021| Autoimmune / Autoinflammatory Disorders

FDA Approves Belumosudil for Chronic Graft-Versus-Host-Disease

The Food and Drug Administration (FDA)  has approved…
Jul 15, 2021| Autoimmune / Autoinflammatory Disorders, Gastrointestinal Diseases

What is Eosinophilic Esophagitis?

Jonathan Rigby, MD, President, Group CEO, and Board…
Jul 14, 2021| Cancers, Hematologic Disorders

Overview of Waldenström’s Macroglobulinemia

Paula Ragan, PhD, CEO and President, X4 Pharmaceuticals,…

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Learning Center

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the central nervous systems of young patients.

Fabry Disease Research Highlights

This CME activity highlights the latest information about Fabry disease from WORLDSymposium 2021.

Tenosynovial Giant Cell Tumor (TGCT)

Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath. These rare tumors are sometimes referred to as giant cell tumor of the tendon sheath (GCT-TS) and/or pigmented villonodular synovitis (PVNS).

Mucopolysaccharidosis I CME Program

Paul Orchard, MD from the University of Minnesota Medical School provides an overview of Mucopolysaccharidosis I in these CME and CNE courses. Dr. Orchard discusses the importance of newborn screening and genetic counseling, and the challenges of diagnosing and treating patients with MPS I.

WHIM Syndrome Learning Center

WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages. However, due to the heterogeneous presentation of the disease, coupled with lack of awareness of the condition, the diagnosis may be delayed, sometimes even into adulthood.

Current and Emerging Therapies for Lysosomal Storage Disease

Drs. Goker-Alpan and Zimran provide an overview of current and emerging therapies for lysosomal storage diseases in this CME program.

Gaucher Disease Research Highlights

This CME activity highlights the latest information about Gaucher disease and provides expert analysis of its clinical relevance.

CME Program: Pulmonary Arterial Hypertension (PAH) Highlights

Richard N Channick, MD highlightsnew information and data regarding pulmonary arterial hypertension (PAH) to assist in the diagnosis and management of patients with this rare disease.

Niemann–Pick Disease Type C Learning Center

Niemann-Pick disease type C (NPC) is a disabling, lysosomal storage disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood.

Angelman Syndrome Learning Center

Angelman syndrome is a rare genetic disorder affecting roughly 500,000 people worldwide. Patients with Angelman syndrome generally experience developmental delays, intellectual disabilities, extensive speech impairment, issues with movement and balance, epilepsy, and abnormal sleep-wake.

CME Program: Eye on Neuromyelitis Optica Spectrum Disorder

Michael Levy, MD, PhD and Prem Subramanian, MD, PhD highlight the important role ophthalmologists and neurologists play in diagnosing and treating NMOSD most efficiently.

Cutaneous T-Cell Lymphoma Learning Center

Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders. It is a rare group of malignancies, with an incidence of 6.4 cases per 1 million people.

CME Program on Tumor-induced Osteomalacia (TIO): Diagnosis and Management

Peter Tebben, MD discusses tumor-induced osteomalacia (TIO), a rare paraneoplastic condition commonly characterized by bone pain, muscle weakness and fractures. 

Hereditary Angioedema Research Highlights: CME Program

Timothy Craig, FAAAAI, FACP, FACOI from Penn State University highlights the latest information about HAE and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with this rare disease.

CME Program: Narcolepsy Research Highlights

Maurice Ohayon, MD, PhD from Stanford University provides highlights of the latest information about narcolepsy along with expert analysis of its potential clinical relevance for busy members of the care team in order to help them care for patients with this rare disease.

Neuroendocrine Tumors (NETs) Research Highlights: CME Program

Edward M Wolin, MD from the Tisch Cancer Institute at Mount Sinai and Icahn School of Medicine at Mount Sinai, highlights the latest information about NETs and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with these rare diseases.

Disease Categories

Topics

Niemann–Pick Disease Type C Learning Center

Niemann-Pick disease type C (NPC) is a disabling, lysosomal storage disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood.

CME Program on Tumor-induced Osteomalacia (TIO): Diagnosis and Management

Peter Tebben, MD discusses tumor-induced osteomalacia (TIO), a rare paraneoplastic condition commonly characterized by bone pain, muscle weakness and fractures. 

Tenosynovial Giant Cell Tumor (TGCT)

Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath. These rare tumors are sometimes referred to as giant cell tumor of the tendon sheath (GCT-TS) and/or pigmented villonodular synovitis (PVNS).

Current and Emerging Therapies for Lysosomal Storage Disease

Drs. Goker-Alpan and Zimran provide an overview of current and emerging therapies for lysosomal storage diseases in this CME program.

Neuroendocrine Tumors (NETs) Research Highlights: CME Program

Edward M Wolin, MD from the Tisch Cancer Institute at Mount Sinai and Icahn School of Medicine at Mount Sinai, highlights the latest information about NETs and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with these rare diseases.

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Learning Center

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the central nervous systems of young patients.

Gaucher Disease Research Highlights

This CME activity highlights the latest information about Gaucher disease and provides expert analysis of its clinical relevance.

Mucopolysaccharidosis I CME Program

Paul Orchard, MD from the University of Minnesota Medical School provides an overview of Mucopolysaccharidosis I in these CME and CNE courses. Dr. Orchard discusses the importance of newborn screening and genetic counseling, and the challenges of diagnosing and treating patients with MPS I.

Fabry Disease Research Highlights

This CME activity highlights the latest information about Fabry disease from WORLDSymposium 2021.

WHIM Syndrome Learning Center

WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages. However, due to the heterogeneous presentation of the disease, coupled with lack of awareness of the condition, the diagnosis may be delayed, sometimes even into adulthood.

CME Program: Pulmonary Arterial Hypertension (PAH) Highlights

Richard N Channick, MD highlightsnew information and data regarding pulmonary arterial hypertension (PAH) to assist in the diagnosis and management of patients with this rare disease.

CME Program: Eye on Neuromyelitis Optica Spectrum Disorder

Michael Levy, MD, PhD and Prem Subramanian, MD, PhD highlight the important role ophthalmologists and neurologists play in diagnosing and treating NMOSD most efficiently.

Angelman Syndrome Learning Center

Angelman syndrome is a rare genetic disorder affecting roughly 500,000 people worldwide. Patients with Angelman syndrome generally experience developmental delays, intellectual disabilities, extensive speech impairment, issues with movement and balance, epilepsy, and abnormal sleep-wake.

Hereditary Angioedema Research Highlights: CME Program

Timothy Craig, FAAAAI, FACP, FACOI from Penn State University highlights the latest information about HAE and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with this rare disease.

CME Program: Narcolepsy Research Highlights

Maurice Ohayon, MD, PhD from Stanford University provides highlights of the latest information about narcolepsy along with expert analysis of its potential clinical relevance for busy members of the care team in order to help them care for patients with this rare disease.

Cutaneous T-Cell Lymphoma Learning Center

Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders. It is a rare group of malignancies, with an incidence of 6.4 cases per 1 million people.