Rare Disease

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FDA Approved | Image credit: B-design - stock.adobe.com

FDA Approves First RNA-Targeted Treatment for Hereditary Angioedema

August 21st 2025

The approval makes donidalorsen the only treatment in the US for hereditary angioedema that is an RNA-targeted prophylactic.

FDA headquarters | Image Credit: Tada Images - stock.adobe.com

FDA Issues CRL for Vatiquinone in Friedreich Ataxia, Requests More Efficacy Data

By Brooke McCormick

August 19th 2025

While the findings suggest that switching to onasemnogene abeparvovec after non–gene therapy treatments could be beneficial to patients with spinal muscular atrophy, it is not clear which patients may benefit the most from switching therapies. | Image credit: Rochu_2008 - stock.adobe.com

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

By Rose McNulty

August 15th 2025

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

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

More News

January 17th 2017

Nearly Half of New Drugs Approved in 2016 Were Orphan Drugs

Laura Joszt, MA

Among the 9 new orphan drugs approved by the FDA in 2016 were 3 treatments for rare diseases that, so far, had no approved treatments: Duchenne muscular dystrophy, spinal muscular atrophy, and severe hepatic veno-occlusive disease.

January 16th 2017

The Value of Survival Gains in Myelodysplastic Syndromes

Joanna P. MacEwan, PhD Wes Yin, PhD Satyin Kaura, MSci, MBA Zeba M. Khan, PhD

This study measured the value of survival gains attributable to the introduction of 3 novel therapies for myelodysplastic syndromes.

January 16th 2017

Cytokine Biomarkers Can Predict Response to CAR T-Cell Treatment in CLL

Surabhi Dangi-Garimella, PhD

At the 58th American Society of Hematology Annual Meeting & Exposition, Jan Joseph Melenhorst, PhD, presented results of a study evaluating biomarkers of response to anti-CD19 CAR T-cell treatment in patients diagnosed with chronic lymphocytic leukemia (CLL).

January 16th 2017

CAR-T Cells of Defined Composition Effective in Ibrutinib-Refractory CLL

Surabhi Dangi-Garimella, PhD

In high-risk patients with chronic lymphocytic leukemia (CLL), CD19 chimeric antigen receptor (CAR)-T cells of defined composition can be administered with an acceptable early toxicity.

January 14th 2017

Initiative to Aid the Fight Against Rare Diseases

AJMC Staff

A new initiative has been launched at the University of North Carolina to create and share tools to fight rare diseases.

January 10th 2017

Reducing GVHD Following Transplant in Lymphoma

Surabhi Dangi-Garimella, PhD

Researchers from the Memorial Sloan Kettering Cancer Center have developed a novel method using modified donor T cells to make stem cell transplants both safer and more effective for treating blood cancers

January 4th 2017

CAR-T Treatment in Glioblastoma: Will the Effect Be Sustained?

Surabhi Dangi-Garimella, PhD

A new single-patient case study, published in the New England Journal of Medicine, has raised the possibility of using CAR-T cells in the treatment of glioblastoma, an aggressive form of brain tumor.

January 3rd 2017

First Patient Treated With Jakafi in Phase 2 Graft-Versus-Host Disease Study

AJMC Staff

The first patient has been treated with ruxolitinib (Jakafi), by Incyte Corporation, for steroid-refractory acute graft-versus-host disease, as part of the REACH-1 phase 2 trial. The trial is evaluating ruxolitinib in combination with corticosteroids.

January 1st 2017

The High Cost of Home Care for Children With Special Healthcare Needs

Laura Joszt, MA

Caring at home for children with special healthcare needs brings high costs to families. A study by researchers at the University of Southern California, Boston Children’s Hospital, and RAND Corp., placed the uncompensated medical cost at nearly $36 billion annually.

December 28th 2016

Reducing Radiation Dose in Oropharynx Cancer Can Improve QOL

Surabhi Dangi-Garimella, PhD

A new clinical study to reduce the unwanted side effects of radiation in patients with human papillomavirus (HPV)—positive oropharynx cancers has found that reducing the dose of radiation can maintain the cure rates but avoid some of the late treatment-associated toxicities.

December 23rd 2016

SWOG Trial Results Could Lead to New Standard of Care in Multiple Myeloma

Surabhi Dangi-Garimella, PhD

The Lancet study shows addition of bortezomib significantly improves survival in treatment-naÃ¯ve patients with multiple myeloma.

December 23rd 2016

Precision Treatment Would Benefit Patients With Glioblastoma, Stanford Analysis Finds

Surabhi Dangi-Garimella, PhD

Using angiogenesis inhibitors in patients with glioblastoma that is highly vascularized could yield much improved outcomes, the analysis found.

December 22nd 2016

Rare Disease State Report Card Identifies Opportunities for Policy Improvement

Christina Mattina

A report card published by the National Organization for Rare Disorders issued grades to each state for 7 categories of rare disease policies.

December 21st 2016

Using a Tumor's Genomic Signature to Guide Radiation Therapy Doses

Surabhi Dangi-Garimella, PhD

Researchers at the Moffitt Cancer Center have developed a genomics model called genomic-adjusted radiation dose that can guide the radiation dose to match a tumor’s radiosensitivity.

December 16th 2016

Craig Sager Loses His Fight Against AML

Surabhi Dangi-Garimella, PhD Mary Caffrey

Craig Sager, a sideline reporter for CNN and Turner Sports, has died a little over 2 and a half years after being diagnosed with acute myeloid leukemia (AML).

December 6th 2016

CAR T-Cells of Defined Composition Effective in Ibrutinib-Refractory CLL

Surabhi Dangi-Garimella, PhD

In high-risk patients with chronic lymphocytic leukemia (CLL), CD19 chimeric antigen receptor (CAR) T-cells of defined composition can be administered with an acceptable early toxicity.

December 5th 2016

Innovative Approach to Precision Trial Design: NCI-MATCH and Beat AML

Surabhi Dangi-Garimella, PhD

Representatives from the Beat acute myeloid leukemia (AML) and National Cancer Institute-Molecular Analysis for Therapy Choice (NCI-MATCH), which incorporate genomic profiling to assign patients to different treatment arms, provided an insight on trial design and a progress report.

December 5th 2016

RESONATE-2 Continues to Impress With Single-Agent Ibrutinib for CLL/SLL at 29 Months

Surabhi Dangi-Garimella, PhD

Older patients diagnosed with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) continue to present a favorable response to single-agent ibrutinib, at a follow-up of 29 months, in phase 3 of the RESONATE-2 trial.

December 3rd 2016

Early Results Show Palbociclib Helps Sustain Patient Response to Ibrutinib in MCL

Surabhi Dangi-Garimella, PhD

Early phase 1 results show that including the cyclin-dependent kinase 4/6 inhibitor palbociclib in the treatment of patients with mantle cell lymphoma (MCL) can help overcome resistance to ibrutinib.

December 3rd 2016

Dr David Porter Compares Use of CAR T-Cells for B-Cell and Solid Tumors

Chimeric antigen receptor (CAR) T-cells have been dramatically effective in treating B-cell cancers, according to David L. Porter, MD, of the University of Pennsylvania Health System. He also identified the use of CAR T-cells for treating solid tumors as a research area that will see more development in the coming years.

December 1st 2016

Precision CAR-T Trial Shows Early Signs of Success in Multiple Myeloma

Surabhi Dangi-Garimella, PhD

Interim data analysis of a phase 1 trial of chimeric antigen receptor-T cells (CAR-T) targeting the B-cell maturation antigen in heavily pretreated patients with multiple myeloma has identified an objective anti-tumor response, with limited toxicity.

November 30th 2016

Statins Could Reduce Mortality in Patients With Multiple Myeloma

Surabhi Dangi-Garimella, PhD

Statin use was associated with a reduction in all-cause and multiple myeloma-specific mortality.

November 25th 2016

Risk Factors for Neutropenia and Infections in Childhood ALL

AJMC Staff

A study published in the Annals of Oncology has analyzed data on young patients with acute lymphoblastic leukemia (ALL) to evaluate risk factors for febrile neutropenia and infection-related complications.

November 23rd 2016

Another Death Reported in Juno's CAR-T Trial in Patients With ALL

AJMC Staff

Within 4 months of the last reported incident, Juno Therapeutics has again halted the phase 2 “Rocket” trial of JCAR015 in patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL).

November 17th 2016

ICU Admission Reduces Survival, Augments Costs Among Patients With AML

Surabhi Dangi-Garimella, PhD

A study published by researchers from the Fred Hutchinson Cancer Research Center in Seattle has found that admission to the intensive care unit reduced survival and increased the cost of care among patients undergoing treatment for acute myeloid leukemia.

November 16th 2016

Online Support Tools Teach Self-Management for Teens With Juvenile Arthritis

Christina Mattina

At the Annual Meeting of the American College of Rheumatology in Washington, DC, several experts presented their progress in harnessing technology to help teenagers and young adults manage their juvenile arthritis.

November 14th 2016

Genes, the Environment, and Autoimmune Disease

David Hafler, MD, chairman of the Department of Neurology at the Yale School of Medicine, spoke of exciting times in the field of genome-wide association study-specifically speaking to neurology, genetics, the environment, and the autoimmune response.

October 31st 2016

Advice for Patients With Atopic Dermatitis

October 31st 2016

Atopic Dermatitis: One Patient's Experience With Instructions Regarding Use of Dupilumab

October 31st 2016

Atopic Dermatitis: Dupilumab and Increase in Patient Quality of Life

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