Rare Disease

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

May 23rd 2025

Explore 5 rare genetic and neurological disorders named after pioneering women in medicine, highlighting their significant contributions to previously unknown conditions.

RAre disease ribbon-Chinnapong-stock.adobe.com
5 Rare Diseases That Now Have Their First FDA-Approved Treatments

February 28th 2025

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

More News

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August 8th 2014

WHO: Ebola Outbreak Is International Public Health Emergency

The World Health Organization declared an international public health emergency Friday over the Ebola outbreak in western Africa that has killed almost 1,000 people. The outbreak of the deadly virus is "extraordinary event" and a public health risk to other countries, it said.
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August 7th 2014

Mutation in a BRCA2-Related Gene: Increased Risk of Breast Cancer

The study, published in NEJM, proposes enhanced surveillance in women with PALB2 mutations, similar to women harboring BRCA2 mutations.
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August 4th 2014

AbbVie's Investigational Compound Receives Orphan Drug Designation by Both the FDA and EMA

ABT-414 is being developed to treat glioblastoma multiforme.
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July 3rd 2014

FDA approves Beleodaq to treat rare, aggressive form of non-Hodgkin lymphoma

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June 27th 2014

Using Clinically Nuanced Cost Sharing to Enhance Consumer Access to Specialty Medications

Jason Buxbaum, MHSA Jonas de Souza, MD A. Mark Fendrick, MD
In conjunction with provider-oriented payment reform, tools like VBID that change the focus from how much to how well we spend our healthcare dollars.
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June 24th 2014

Drug Discount Program Has Drugmakers Crying Foul

This spring as the feds have been drawing up new rules for the program, a pitched battle has broken out between hospitals and drug manufacturers who say the program, known as 340B, is now bloated and badly regulated.
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June 19th 2014

As Sequencing Moves into Clinical Use, Insurers Balk

Once strictly the domain of research labs, gene-sequencing tests increasingly are being used to help understand the genetic causes of rare disease, putting insurance companies in the position of deciding whether to pay the $5,000 to $17,000 for the tests.
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May 28th 2014

Stakeholders Weigh-In on Excessive Drug Costs

Katie Sullivan, MA
Recent controversy over the cost of Sovaldi (Gilead Sciences)-a $1000-per-dose treatment for hepatitis C-has sparked a debate as to whether there should be federal caps on the costs of certain prescription drugs.
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April 22nd 2014

Evolution of Superbugs: A Healthcare Nightmare

Surabhi Dangi-Garimella, PhD
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March 13th 2014

New Orphan Drug in the T1DM Realm: Another Immunotherapy Success Story?

Surabhi Dangi-Garimella, PhD
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March 3rd 2014

Chronically Ill Face High Out-of-Pocket Costs

Katie Sullivan
Those with chronic illnesses such as leukemia, hepatitis C, and multiple sclerosis may face a significant increase in their out-of-pocket expenses for specialty drugs. This is because payers are replacing fixed-dollar copayments with coinsurance rates that require patients to contribute a higher percentage of the cost for their specialty medications.
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February 20th 2014

The Impact of Patient Assistance Programs and the 340B Drug Pricing Program on Medication Cost

Yelba M. Castellon, MD Shahrzad Bazargan-Hejazi, PhD Miles Masatsugu, MD Roberto Contreras, MD
Examining the financial impact that patient assistance programs and the 340B Drug Pricing Program have on improving medication cost.
Opportunities in Utilization Management: Making Specialty Drugs Affordable
February 4th 2014

Opportunities in Utilization Management: Making Specialty Drugs Affordable

When it comes to managing chronic conditions and rare disease, the cost of treatment can be astronomical.
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October 18th 2013

Managing Rare Disease and Orphan Drug Costs

Katie Sullivan
The cost to treat rare disease can be extraordinary.
Fatigue and Cognitive Impairment in Multiple Sclerosis
October 4th 2013

Fatigue and Cognitive Impairment in Multiple Sclerosis

Fatigue and cognitive impairment are 2 of the most common health problems associated with multiple sclerosis (MS). Recent MS studies evaluate the nature of these 2 conditions not just as side effects of MS, but as chronic conditions with varying degrees of severity based on objective assessment and self-perception.
Episode 15 - SLE: Expectations As the Drug Market Evolves
July 23rd 2013

Episode 15 - SLE: Expectations As the Drug Market Evolves

Episode 19 - The Future of SLE and Hemophilia
July 23rd 2013

Episode 19 - The Future of SLE and Hemophilia

Episode 18 - The Role of Specialty Pharmacies in Rare Diseases
July 23rd 2013

Episode 18 - The Role of Specialty Pharmacies in Rare Diseases

Episode 17 - Future Outlook on the Orphan Drug Market
July 23rd 2013

Episode 17 - Future Outlook on the Orphan Drug Market

Episode 16 - Factors Hindering Care for SLE Patients
July 23rd 2013

Episode 16 - Factors Hindering Care for SLE Patients

Episode 13 - Challenges of Measuring the Outcomes of SLE
July 23rd 2013

Episode 13 - Challenges of Measuring the Outcomes of SLE

Episode 14 - Unmet Needs for Treatment of SLE
July 23rd 2013

Episode 14 - Unmet Needs for Treatment of SLE

Episode 12 - Recognizing the Strengths and Weaknesses of Current Clinical Practice Guidelines
July 23rd 2013

Episode 12 - Recognizing the Strengths and Weaknesses of Current Clinical Practice Guidelines

Episode 11 - Minimizing the Burden of SLE
July 23rd 2013

Episode 11 - Minimizing the Burden of SLE

Episode 10 - Treating Mild and Severe Hemophilia
July 23rd 2013

Episode 10 - Treating Mild and Severe Hemophilia

Episode 9 - The Future Landscape for the Treatment of Hemophilia
July 23rd 2013

Episode 9 - The Future Landscape for the Treatment of Hemophilia

Episode 8 - Long-Acting Agents and Considerations
July 23rd 2013

Episode 8 - Long-Acting Agents and Considerations

Episode 7 - Minimizing the Burden of SLE
July 23rd 2013

Episode 7 - Minimizing the Burden of SLE

Episode 6 - Addressing the Gaps in Care for Hemophilia Patients
July 23rd 2013

Episode 6 - Addressing the Gaps in Care for Hemophilia Patients

Episode 5 - Current Therapeutic Strategies for Hemophilia
July 23rd 2013

Episode 5 - Current Therapeutic Strategies for Hemophilia

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