Rare Disease

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

February 28th 2025

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease.

FDA approved. | Image Credit: Pawel - stock.adobe.com
FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas

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

November 15th 2024

Patients with SMA and their caregivers stand to benefit from shared decision-making processes | image credit: Intelligent Horizons - stock.adobe.com
Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments

November 8th 2024

SMA type is a strong predictor of some oral functioning in affected patients | image credit: RTimages - stock.adobe.com
Oral Function Impairment Varies Across Adult Patients With SMA

October 28th 2024

More News

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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.
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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.
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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-naïve patients with multiple myeloma.
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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.
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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.
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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.
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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).
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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.
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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.
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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.
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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.
Dr David Porter Compares Use of CAR T-Cells for B-Cell and Solid Tumors
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.
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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.
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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.
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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.
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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).
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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.
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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.
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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.
Advice for Patients With Atopic Dermatitis
October 31st 2016

Advice for Patients With Atopic Dermatitis

Atopic Dermatitis: One Patient's Experience With Instructions Regarding Use of Dupilumab
October 31st 2016

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

Atopic Dermatitis: Dupilumab and Increase in Patient Quality of Life
October 31st 2016

Atopic Dermatitis: Dupilumab and Increase in Patient Quality of Life

Impact of Atopic Dermatitis on Day-to-Day Life
October 31st 2016

Impact of Atopic Dermatitis on Day-to-Day Life

One Patient's Journey With Atopic Dermatitis
October 31st 2016

One Patient's Journey With Atopic Dermatitis

The Future of Atopic Dermatitis
October 31st 2016

The Future of Atopic Dermatitis

Specialty Pharmacy, Payer Goals in Atopic Dermatitis Management
October 31st 2016

Specialty Pharmacy, Payer Goals in Atopic Dermatitis Management

Atopic Dermatitis: Evolving Role of Specialty Pharmacy
October 31st 2016

Atopic Dermatitis: Evolving Role of Specialty Pharmacy

Utilization Management Nuances in Atopic Dermatitis
October 31st 2016

Utilization Management Nuances in Atopic Dermatitis

Practical Application of Clinical Trial Measures in Atopic Dermatitis
October 31st 2016

Practical Application of Clinical Trial Measures in Atopic Dermatitis

Evidence and Outcomes in Atopic Dermatitis
October 31st 2016

Evidence and Outcomes in Atopic Dermatitis

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