Rare Disease

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Dordaviprone is indicated for recurrent H3 K27M-mutant diffuse midline glioma, an ultra-rare, aggressive brain tumor primarily affecting children. | Image credit: wladimir1804 - stock.adobe.com

FDA Approves Dordaviprone as First and Only Treatment for Aggressive Form of Glioma

By Rose McNulty

August 6th 2025

Dordaviprone (Modeyso) is now approved to treat recurrent H3 K27M-mutant diffuse midline glioma, an ultra-rare, aggressive brain tumor primarily affecting children.

Women in science | Image Credit: © metamorworks-stock.adobe.com.jpeg

5 Rare Disorders Named for Pioneering Female Scientists

By Maggie L. Shaw

May 23rd 2025

RAre disease ribbon-Chinnapong-stock.adobe.com

5 Rare Diseases That Now Have Their First FDA-Approved Treatments

By Giuliana Grossi

February 28th 2025

FDA approved. | Image Credit: Pawel - stock.adobe.com

FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas

By Giuliana Grossi

February 12th 2025

Gene therapy, gene editing concept | image credit: vchalup - stock.adobe.com

Delandistrogene Moxeparvovec Fails to Meet Primary End Point in Phase 3 Study

By AJMC Contributor

November 15th 2024

More News

July 28th 2017

Breakthrough Therapy Designation for Venetoclax in AML

Venetoclax (Venclexta), in combination with low dose cytarabine, has been granted a breakthrough therapy designation for use in elderly, treatment-naive patients with acute myeloid leukemia (AML) who cannot handle intensive chemotherapy.

July 26th 2017

Patient Questionnaires Can Predict Clinical Outcomes in Patients With Multiple Sclerosis

Alison Rodriguez

Patient-reported outcomes (PROs) can demonstrate the effects of a disease or treatment. By using patient questionnaires, the results can be analyzed to predict clinical outcomes in medical research for diseases, including multiple sclerosis (MS).

July 25th 2017

Mechanism for Checkpoint Inhibitor Resistance Identified in Invasive Bladder Cancer

Christina Mattina

Researchers have identified a cellular signaling pathway that when activated by mutations may prevent immune cells from infiltrating bladder tumors, thus making the cancer resistant to immune checkpoint inhibitors.

July 25th 2017

What We're Reading: Monthly HIV Shot; Molina Layoffs; Charlie Gard Treatment

July 20th 2017

Rare Diseases Create Significant Care and Financial Burdens Among Patients

July 19th 2017

Nivolumab, Alone or With Ipilimumab, Met the Disease Control Rate in Malignant Pleural Mesothelioma

In a late-breaking abstract at the 2017 American Society of Clinical Oncology Annual Meeting, researchers presented an interim analysis of a randomized phase 2 trial of second- or third-line nivolumab, with or without ipilimumab, in patients with second- or third-line malignant pleural mesothelioma.

July 18th 2017

New Report Examines the Biopharmaceutical Pipeline and Innovation in Clinical Development

Alison Rodriguez

Report notes that 74% of drugs that are currently in development are potentially first-in-class medicines, and 822 drugs in development are for rare diseases.

July 17th 2017

Daratumumab-Based Combination Effective in Relapsed/Refractory Multiple Myeloma, Short PFS in Heavily Pretreated Patients

Surabhi Dangi-Garimella, PhD

Managing heavily pretreated, often less fit, patients with relapsed/refractory multiple myeloma is a challenge in routine practice, as illustrated by the fact progression-free survival remains short, although daratumumab-based combination therapies are proven effective, according to results presented during a poster session at the 2017 American Society of Clinical Oncology Annual Meeting.

July 17th 2017

Phase 1 Study Results at ASCO Support First-Line Use of Daratumumab in Multiple Myeloma

Surabhi Dangi-Garimella, PhD

A phase 1 study, presented at the 2017 Annual Meeting of the American Society of Clinical Oncology, found that using daratumumab, an antibody that binds and inhibits the CD38 receptor, can improve patient response to treatment.

July 17th 2017

Daratumumab With Standard Regimen Improves PFS, ORR Independent of Cytogenetic Risk in Multiple Myeloma

Surabhi Dangi-Garimella, PhD

Updated trial results at the 2017 Annual Meeting of the American Society of Clinical Oncology showed the combinations of daratumumab with either lenalidomide and dexamethasone or bortezomib and dexamethasone prolonged progression-free survival for patients with relapsed refractory multiple myeloma.

July 14th 2017

Investor Reactions to Orphan Drug Designation Strongest for Oncology Therapies, Study Finds

Alison Rodriguez

A new study examines the impact that a pharmaceutical’s designation as an orphan drug can have on its manufacturer’s stock prices, and finds that the largest returns were for oncology drugs.

July 12th 2017

FDA Grants Full Approval, Expands Indications for Blinatumomab in ALL

Christina Mattina

Blinatumomab is now fully approved as a treatment for relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), following FDA assessment of new trial data on survival outcomes and its use in certain clinical subgroups.

July 12th 2017

CHESS Study Investigates Economic Burden of Severe Hemophilia in Europe

Alison Rodriguez

Severe hemophilia often results in a significant economic and psychological burden on patients, caregivers, and the healthcare system as a whole, according to recent research from Europe.

July 12th 2017

Genome Sequencing Reveals Rare Disease Risk in 1 of 5 Generally Healthy Adults

Laura Joszt, MA

An analysis of whole-genome sequencing (WGS) in healthy adults in the primary care setting found that 1 in 5 had a previously unrecognized variant associated with a rare disease, according to a study published in Annals of Internal Medicine.

July 11th 2017

Novartis' CAR-T Therapy for Pediatric Leukemia to Face FDA Advisory Panel

Alison Rodriguez

Novartis’ chimeric antigen receptor T-cell (CAR-T) therapy for treating pediatric leukemia is on the cusp of being the first FDA-approved gene therapy, which will lead to new developments and utilizations of CAR-T therapy for treating other advanced blood cancers.

July 9th 2017

New Antibody Demonstrates 2-Pronged Approach in Treating Blood Cancer

Alison Rodriguez

A study published by BloodAdvances, a Journal of the American Society of Hematology, demonstrated the potential of the 2-pronged approach in treating non-Hodgkin lymphoma, multiple myeloma, and acute myeloid leukemia.

July 5th 2017

Sarepta Therapeutics and Genethon Announce DMD Treatment Research Collaboration

Alison Rodriguez

Sarepta Therapeutics has announced a new collaboration with Genethon for gene therapy research in efforts to develop new Duchenne muscular dystrophy (DMD) treatments.

July 3rd 2017

Rare Disease Can Be Misdiagnosed as Rheumatoid Arthritis

Laura Joszt, MA

Whipple’s disease (WD) is a rare disease that predominantly affects middle-aged white men, and its diagnosis is often delayed because it is misdiagnosed as rheumatoid arthritis. A new study in Orphanet Journal of Rare Diseases aimed to describe clinical symptoms of WD and appropriate treatment.

June 29th 2017

Novel Antibody May Restore Immune Response to Rituximab for CLL

Christina Mattina

Among a group of patients with chronic lymphocytic leukemia (CLL) who were previously unresponsive to rituximab, a new antibody could help enable the immune system to attack the cancer cells, according to a study in PLOS One.

June 28th 2017

Nivolumab Improved Patient-Reported QOL Compared With Investigator's Choice: CheckMate 141

Surabhi Dangi-Garimella, PhD

The study, published in Lancet Oncology, has found that nivolumab reduced the rate of clinically meaningful deterioration compared with investigator’s choice among platinum-refractory patients being treated for recurrent or metastatic head and neck cancer.

June 27th 2017

Infusing a Sense of Urgency in Cancer Care: The Biden Cancer Initiative

Surabhi Dangi-Garimella, PhD

On Monday, June 26, 2017, the Biden Foundation announced the launch of the Biden Cancer Initiative, their new venture to continue the fight to make progress in cancer prevention, detection, treatment, and care.

June 27th 2017

Venclexta/Venclyxto Effective in Treating Chronic Lymphocytic Leukemia

Alison Rodriguez

Data from multiple studies have demonstrated the efficacy of Venclexta/Venclyxto in treating relapsed or refractory chronic lymphocytic leukemia, according to Roche.

June 25th 2017

Diagnostic Immunoassays Could Spare Patients High-Risk Pancreatic Resection

Surabhi Dangi-Garimella, PhD

Researchers at Indiana University have confirmed the clinical utility of 2 laboratory tests that can distinguish benign pancreatic cancer lesions that mimic early signs of cancer and spare patients of unnecessary pancreatic cancer screenings or surgeries.

June 24th 2017

FDA Approves Rituxan Hycela for Treating Certain Blood Cancers

Alison Rodriguez

The FDA has approved Genentech’s Rituxan Hycela for subcutaneous injection for the treatment of certain blood cancers. Rituxan Hycela has similar outcomes as Rituxan, but can be delivered in 5 to 7 minutes compared with 1.5 hours for Rituxan.

June 23rd 2017

Genentech's Ocrevus Reduces Disease Progression in Multiple Sclerosis Patients

Alison Rodriguez

A phase 3 clinical trial of patients with relapsing multiple sclerosis and primary progressive multiple sclerosis found that Ocrevus (ocrelizumab) significantly reduced disease progression, according to results released by Genentech.

June 23rd 2017

FDA Approves of Darzalex Combination for Multiple Myeloma

Alison Rodriguez

The FDA has approved the use of Janssen Biotech, Inc’s immunotherapy Darzalex in combination with pomalidomide and dexamethasone. Darzalex is used to treat multiple myeloma patients who have previously received at least 2 therapies with lenalidomide and a proteasome inhibitor.

June 14th 2017

Genome-Wide Association Studies Identify Novel Risk Loci for Testicular Cancer

Surabhi Dangi-Garimella, PhD

A meta-analysis spearheaded by The International Testicular Cancer Consortium has identified new susceptibility loci in the human genome that can increase a person’s risk of developing inherited testicular germ cell tumors.

June 14th 2017

A Drug Unsuccessful at Treating Cancer May Successfully Treat DMD

Alison Rodriguez

A drug previously used in an attempt to treat cancer, now has the potential to be repurposed to treat Duchenne muscular dystrophy.

June 7th 2017

Phase 1 Study Results at ASCO Support First-Line Use of Daratumumab in Multiple Myeloma

A phase 1 study, presented at the 2017 American Society of Clinical Oncology Annual Meeting, found that including daratumumab can improve patient response to treatment in newly diagnosed multiple myeloma.

June 5th 2017

Daratumumab With Standard Regimen Improves PFS in Multiple Myeloma, Independent of Cytogenetic Risk

Surabhi Dangi-Garimella, PhD

Updated results from the POLLUX and CASTOR trials, presented at the 2017 American Society of Clinical Oncology Annual Meeting, have found that including daratumumab in standard-of-care regimens prolonged progression-free survival (PFS) and improved the depth of response, independent of the patients’ cytogenetic risk.

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