Cancers

There are over 200 different types of rare cancers, but only a small number of people are diagnosed with each type. Many of those cancers are highlighted in this section.
Jun 23, 2025| Posted in: Advocacy, Cancers, Diagnosis, Hematologic Disorders, Treatment

Chronic Myeloid Leukemia: Overview, Treatment Options, and Advice

Jorge Cortes, MD, Director of the Georgia Cancer…
Jun 19, 2025| Posted in: Cancers, Drug Development, Hematologic Disorders

Linvoseltamab Combination Therapy in Patients With Relapsed/Refractory Multiple Myeloma

Salomon Manier, MD, PhD, Associate Professor of Hematology…
Jun 13, 2025| Posted in: Cancers, Lung Diseases, Regulations, Treatment

FDA Approves Taletrectinib for Rare Form of Lung Cancer

The U.S. Food and Drug Administration (FDA) has…
Jun 10, 2025| Posted in: Advocacy, Cancers, Hematologic Disorders, Lung Diseases

Diagnostic Odyssey With Histiocytosis

Nate Milam II, Patient Advocate for the Histiocytosis…
Jun 3, 2025| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Diagnosis, Hematologic Disorders

A New Test for Patient-Reporting of Mastocytosis Control

A team of allergists, immunologists, and dermatologists from…

Recent Videos

Cutaneous T-Cell Lymphoma

Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders.[1] Cutaneous T-cell lymphoma is a rare group of malignancies, with an incidence of 6.4 cases per 1 million people. This form of T-cell lymphoma represents around 70% of primary cutaneous lymphomas.

Optimizing Therapeutic Proteins Through PEGylation: Key Parameters and Impacts

The session will delve into the process of PEGylation, where polyethylene glycol (PEG) is conjugated to functional amino acid groups on the protein surface. This modification may enhance the properties of therapeutic proteins, offering advantages in stability, half-life, and immunogenicity.

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).[1-5]

Neuroblastoma

Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. It is a neuroendocrine tumor that originates in neuroblasts or neural crest progenitor cells.

Cancers

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Robert Mitchell-Thain, chair of the PBC Foundation, discusses how linerixibat reduces itching in patients with primary biliary cholangitis (PBC).

https://checkrare.com/linerixibat-reduces-itching-in-patients-with-pbc/

#CheckRare #RareDisease #RareGastrointestinal #RareAutoimmune

Has your patient been diagnosed with immunoglobulin A nephropathy (IgAN), or Berger’s disease? They may be eligible to take part in a new clinical research study evaluating an investigational treatment option that may help manage and slow disease progression. See if they may be eligible to help ...advance research for the IgAN community. Click here to see if they may qualify.

https://icanclinicalstudy.alexionclinicaltrials.com/?utm_source=CheckRare&utm_medium=Social&utm_content=Awareness&utm_campaign=ICAN

Leticia Orsatti, MD, discusses results from clinical trials examining the safety and efficacy of nerandomilast to treat patients with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF).

...https://checkrare.com/evaluating-nerandomilast-in-patients-with-rare-pulmonary-fibrotic-conditions/

#CheckRare #RareLung #RareDisease

The FDA has recently approved the following.

Stay up to date with upcoming PDUFA dates at https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/

#CheckRare #RareDisease #FDAApprovals

The FDA Priority Review Voucher (PRV) program aims to incentivize the development of drugs for rare pediatric and tropical diseases. PRVs act as a way to skip to the front of the FDA’s line to have a future drug reviewed.

Click here to visit CheckRare’s chart tracking issued PRVs and ...their current status https://checkrare.com/priority-review-voucher/

#CheckRare #RareDisease #PriorityReview

Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath.

Learn more at https://checkrare.com/tenosynovial-giant-cell-tumor-tgct/

#CheckRare #RareDisease #TGCT

Matt Winton, PhD, Senior Vice President and Chief Operating Officer of Inozyme Pharma, discusses results from an analysis characterizing disease severity and progression in patients with ENPP1 deficiency.

...https://checkrare.com/disease-severity-and-progression-in-patients-with-enpp1-deficiency/

#CheckRare #ENPP1 #RareMetabolic #RareGenetic

New Data From EMBARK Study in Patients With Duchenne Muscular Dystrophy Receiving Gene Therapy

NEW: Check out our Rare Disease Recognition and Awareness Days page to stay up to date on the latest rare disease awareness programs.

https://checkrare.com/rare-disease-awareness-days-and-recognition-page/

#CheckRare #RareDisease #RareDiseaseAwareness

Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).

https://checkrare.com/a-familys-experience-with-alsp/

#CheckRare #ALSP #RareNeurology ...#RareGenetic

Genetic Testing for WHIM Syndrome: reducing barriers, proper panel analysis, and how to deal with variants of uncertain significance.

Learn more at https://checkrare.com/genetic-testing-for-whim-syndrome/

#CheckRare #WHIMSyndrome #RareGenetic #RareAutoimmune #RareSkin

💡Rare Disease Spotlight: SYNGAP1-Related Disorders

Learn more about this rare disease with our latest article https://checkrare.com/the-current-landscape-of-syngap1-related-disorders/

#CheckRare #RareDisease #SYNGAP1 #RareNeurology #RareGenetic

📢Tomorrow, June 10th at 1:00pm EST

Join us for a Live CME Webinar on Skeletal Involvement in Lysosomal Disorders with Ozlem Goker-Alpan, MD, and Ravi Kamath, MD, PhD.

Click the link to sign up: https://us06web.zoom.us/webinar/register/WN_fCXmUAdMTdysE_HnDniVJw#/registration
...
#CheckRare #LiveCME #RareDisease