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.
April 29th 2024
FDA approves mavorixafor (XOLREMDI) for WHIM syndrome, the first approved treatment for the rare disease.
FDA Accepts IND and Grants Fast Track Status to Sanfilippo Drug
January 23rd 2018This morning, Sobi, announced that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for sobi003, and the regulatory agency granted Fast Track status to the product candidate.
Therapy for Difficult-to-Treat Skin Disease Now Available in Japan
Regeneron has announced that Dupixent, a therapy for adult atopic dermatitis (AD) that is inadequately controlled by existing medications, has been granted marketing and manufacturing authorization in Japan.
Cholangiocarcinoma: Clinical Trials
January 17th 2018In this Rare Connections program, Martha Raymond; Founder of the Raymond Foundation, Executive Director of Michael’s Mission, and the Co-Chair of the GI Cancers Alliance meets with Sarah Bennett, a cholangiocarcinoma survivor to highlight the space.
FDA Gives Priority Review to Kymriah for Second Rare Cancer Indication
January 17th 2018This morning, Novartis was granted U.S. FDA Priority Review for the drug in patients with relapsed or refractory diffuse large B-cell lymphoma who are ineligible for or relapse after autologous stem cell transplant (ASCT).
FDA Accepts BLA for Potential HAE Prophylaxis Therapy
January 17th 2018The FDA has accepted for review supplemental Biologics License Application for Ruconest for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema, Pharming Group N.V. reported this morning.
Cholangiocarcinoma: Care Teams and Patient Support
January 16th 2018In this Rare Connections program, Martha Raymond; Founder of the Raymond Foundation, Executive Director of Michael’s Mission, and the Co-Chair of the GI Cancers Alliance meets with Sarah Bennett, a cholangiocarcinoma survivor to highlight the space.
St. Jude Develops Gene Therapy for Severe Combined Immunodeficiency
Researchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency, commonly referred to as the “Bubble Boy” disease.
Afatinib Approval Expanded for Rare Lung Cancer Treatment
January 15th 2018Boehringer Ingelheim announced the approval of a new indication for afatinib, as the U.S. FDA approved a supplemental NDA for the first-line treatment of patients with metastatic NSCLC whose tumors have non-resistant EGFR mutations.