Latest Summaries

Sep 24, 2021| Hematologic Disorders, News, Regulations, Treatment

FDA Approves Ruxolitinib to Treat Chronic GVHD

The FDA has approved ruxolitinib (Jakafi)  to treat…
Sep 21, 2021| Cancers, Drug Development, Hematologic Disorders

Will Cilta-Cell Be the Next CAR T-Cell Approval in Multiple Myeloma?

Saad Usmani, MD at Levine Cancer Institute discusses…
Sep 17, 2021| Neurology/Nervous System Diseases

What is Lennox-Gastaut Syndrome?

Tracy Dixon-Salazar, PhD, Executive Director of the Lennox-Gastaut…
Sep 14, 2021| Drug Development, Neurology/Nervous System Diseases

How Sleep Is Measured in a Narcolepsy Clinical Trial

Clete A. Kushida, MD, PhD, Neurologist and Division…
Sep 9, 2021| Cancers, Skin Conditions, Treatment

What Are the Treatment Options for CTCL?

Sima Rozati, MD, Assistant Professor of Dermatology at…
Sep 7, 2021| Cancers, Congenital And Genetic Conditions

Overview of Well-Differentiated and Dedifferentiated Liposarcoma

Avanish Vellanki, Cofounder and CEO at Rain Therapeutics,…
Sep 3, 2021| Cancers, Skin Conditions

CTCL Presentation Varies Widely Among Patients, Even Within Subtypes

Sima Rozati, MD, Assistant Professor of Dermatology at…

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.

Gaucher Disease Research Highlights

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

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.

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).

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.

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.

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.

CME Program: Pulmonary Arterial Hypertension (PAH) Highlights

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

Fabry Disease Research Highlights

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

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 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. 

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.

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.

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.

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.