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

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

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

August 6th 2025

Women in science | Image Credit: © metamorworks-stock.adobe.com.jpeg
5 Rare Disorders Named for Pioneering Female Scientists

May 23rd 2025

More News

Managed Care's Role in Future Treatment of PAH
July 18th 2016

Managed Care's Role in Future Treatment of PAH

PAH Treatment Driven by Data From Peer-Reviewed Literature
July 18th 2016

PAH Treatment Driven by Data From Peer-Reviewed Literature

Hospitalization in PAH
July 18th 2016

Hospitalization in PAH

Optimizing PAH Treatment in Managed Care
July 18th 2016

Optimizing PAH Treatment in Managed Care

PAH: Multidisciplinary Care and Medication Benefits
July 18th 2016

PAH: Multidisciplinary Care and Medication Benefits

Long-Term Outcomes and Managing PAH Costs
July 18th 2016

Long-Term Outcomes and Managing PAH Costs

The Importance of a Patient-Centered Approach in PAH
July 18th 2016

The Importance of a Patient-Centered Approach in PAH

Understanding the Newer Options in PAH
July 18th 2016

Understanding the Newer Options in PAH

Prostacyclins in PAH: Selecting Among the Agents
July 18th 2016

Prostacyclins in PAH: Selecting Among the Agents

Outcomes Assessment and Combination Therapy in PAH
July 18th 2016

Outcomes Assessment and Combination Therapy in PAH

Factors Impacting Formulary Decision-Making in PAH
July 18th 2016

Factors Impacting Formulary Decision-Making in PAH

Incorporating Newer Oral PAH Medications
July 18th 2016

Incorporating Newer Oral PAH Medications

Evolution in Clinical Trial Design: Measuring PAH Treatment Efficacy
July 18th 2016

Evolution in Clinical Trial Design: Measuring PAH Treatment Efficacy

The Effect of Delayed Diagnosis of PAH and Identifying Patients Earlier
July 18th 2016

The Effect of Delayed Diagnosis of PAH and Identifying Patients Earlier

The Underlying Pathophysiology of Pulmonary Arterial Hypertension
July 18th 2016

The Underlying Pathophysiology of Pulmonary Arterial Hypertension

Proxy Endpoints to Understand Outcomes and Guide Treatment in PAH
July 18th 2016

Proxy Endpoints to Understand Outcomes and Guide Treatment in PAH

Value of Early Diagnosis of PAH in Managed Care
July 18th 2016

Value of Early Diagnosis of PAH in Managed Care

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June 29th 2016

Claiming "Orphan" Status, AstraZeneca Sues FDA to Protect Crestor Patent

Mary Caffrey
The lawsuit seeks an additional 7 years' protection for the popular cholesterol drug, which sold 20.3 million prescriptions last year.
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June 11th 2016

Duchenne Muscular Dystrophy Treatments in a State of Flux

Jackie Syrop
Treatment for Duchenne muscular dystrophy remains in flux with the FDA delaying a decision on Sarepta's treatment and Biomarin discontinuing development on its own drug.
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May 26th 2016

Research Emerging That Links Insulin Resistance to Cognitive Decline

Andrew Smith
The earliest studies involving diabetes therapy for Alzheimer’s are recruiting patients.
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May 13th 2016

Significant Toxicity of Mitoxantrone in Patients With Multiple Sclerosis: Cancer

Surabhi Dangi-Garimella, PhD
Researchers in Germany have discovered that mitoxantrone-used to treat aggressive, relapsing, or progressive multiple sclerosis-can increase the risk of leukemia and colorectal cancer in those being treated.
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April 11th 2016

BCL-2 Inhibitor Venetoclax Approved as Second Line for CLL With 17p Deletion

Surabhi Dangi-Garimella, PhD
The approval comes following the review of a phase 2 single-arm trial in 106 patients with chronic lymphocytic leukemia who carried the 17p deletion mutation and who had received at least 1 prior therapy for their condition.
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March 18th 2016

The MS Center of Saint Louis: A Practice Perspective of the Impacts of High Cost Sharing

Katherine Upshur, MSW, LCSW
This case study from the MS Center of Saint Louis examines how patients are impacted by high healthcare costs and addresses potential solutions to this problem.
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March 17th 2016

Impact of Cost Sharing on Specialty Drug Utilization and Outcomes: A Review of the Evidence and Future Directions

Jalpa A. Doshi, PhD Pengxiang Li, PhD Vrushabh P. Ladage, BS Amy R. Pettit, PhD Erin A. Taylor, PhD, MSPH
The authors review published evidence regarding associations between high cost sharing for specialty pharmaceuticals indicated for rheumatoid arthritis, multiple sclerosis, and cancer, and their utilization.
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March 4th 2016

Phase 3 RESONATE-2 Data Yield First-Line Approval for Ibrutinib in CLL

Surabhi Dangi-Garimella, PhD
Just 2 days after being rejected by cost regulators in the UK, ibrutinib has been approved for the frontline treatment of patients with chronic lymphocytic leukemia in the United States.
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March 2nd 2016

NICE Rejects Another Cancer Drug, Stresses Low Cost-Effectiveness

Surabhi Dangi-Garimella, PhD
Imbruvica, developed by Janssen for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia who are unsuitable to receive chemo-immunotherapy, has been rejected by UK’s National Institute of Health and Care Excellence.
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December 9th 2015

Insurance Status Can Compromise Outcomes in CML

Surabhi Dangi-Garimella, PhD
Research presented during a health outcomes session at the recently concluded annual meeting of the American Society of Hematology evaluated the influence of patients’ insurance status on clinical outcomes of CML.
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November 1st 2015

Dementia's End-of-Life Costs Surpass Cancer, Heart Disease

Laura Joszt, MA
During the last 5 years of life, the cost of dementia eclipses that of other diseases, like cancer and heart disease, according to a study from the National Institutes of Health.
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October 16th 2015

NCCN Unveils Value Tool for CML and MM

AJMC Staff
NCCN Evidence Blocks add affordability to Guidelines recommendations on safety, efficacy, quality, and consistency of regimens.
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October 7th 2015

Avelumab Receives Fast-Track for Merkel Cell Carcinoma

AJMC Staff
The PD-L1 inhibitor was granted orphan drug status just last month.
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