Rare Disease

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

FDA Approves First RNA-Targeted Treatment for Hereditary Angioedema

By Julia Bonavitacola

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

September 16th 2015

Alzheimer's Disease Costs Disproportionately Affect Women

Laura Joszt, MA

Women bear greater costs related to Alzheimer's disease (AD) compared with men for 2 reasons: they are at greater risk of developing the AD and cost Medicare and Medicaid more, and they are more likely to provide informal, unpaid care to family members with AD.

September 8th 2015

California Law May Show Limits of How Far Managed Care Can Go

Mary K. Caffrey

Parents and advocates for a slower timetable pointed to existing issues within Medi-Cal as proof that the most vulnerable should not be rushed into managed care. But other states are moving ahead with plans.

August 17th 2015

Parkinson's Disease Psychosis: Symptoms, Management, and Economic Burden

Neal Hermanowicz, MD Kari Edwards, PhD

August 3rd 2015

Misdiagnosis of Alzheimer's Disease Is Costly for Patients

Laura Joszt, MA

The diagnosis and management of patients with dementing illnesses can be challenging, but the cost of misdiagnosing dementia as Alzheimer's disease can be as high as $14,000 a year, according to a study published in Alzheimer's & Dementia.

July 22nd 2015

Patients With Alzheimer's Disease Will Consume a Quarter of Medicare's Spending

Laura Joszt, MA

By 2050 more than 28 million additional baby boomers will develop Alzheimer's disease and the cost of caring for them will account for nearly one-fourth of Medicare spending by 2040, according to research presented at the Alzheimer's Association International Conference.

July 8th 2015

How Do You Manage Care in Cystic Fibrosis Patients?

Surabhi Dangi-Garimella, PhD

Till Kalydeco was approved, cystic fibrosis patients primarily received only disease management support to extend their lives.

June 8th 2015

21st Century Cures Brings Hope for Rare Disease Research

However, not everyone supports the bill's provisions that promote the development of pediatric rare disease drugs through the "voucher" incentive.

May 24th 2015

Germany Hesitates Over the World's First $1 Million Drug

Laura Joszt, MA

German health authorities are debating whether the price of Europe's first approved gene therapy is worth the $1 million price tag.

May 2nd 2015

Speedy Drug Approvals Have Become the Rule, Not the Exception

A majority of recent drug development has been in therapies that qualify for at least one special approval pathway that Congress has passed over the past few decades for important drugs that treat life-threatening or rare diseases.

March 26th 2015

Why Pharma Is Investing in Rare Diseases

As patents on some of the most lucrative medicines began to expire, many companies shifted money to rare-disease drugs, knowing that those medicines cost less to develop and will face limited competition.

March 21st 2015

Biogen's Alzheimer's Drug Sharply Slows Cognitive Decline

An experimental drug for Alzheimer's disease sharply slowed the decline in mental function in a small clinical trial, researchers reported Friday, reviving hopes for an approach to therapy that until now has experienced repeated failures.

January 20th 2015

ASCO Declares CLL Regimens Cancer

In it's report, "Clinical Cancer Advances 2015: ASCO's Annual Report on Progress Against Cancer," published in the Journal of Clinical Oncology, 4 newly approved treatments for CLL were predicted to have a dramatic impact on patients with the disease.

January 19th 2015

Poster Presentations Cover Therapeutic Options in CML

Surabhi Dangi-Garimella, PhD

December 29th 2014

FDA Approves New Ebola Test for Emergency Use

The FDA has provided Emergency Use Authorization, a pre-approval for use in bad outbreaks, for Roche's new Ebola test, the LightMix Ebola Zaire rRT-PCR Test.

December 9th 2014

SPIRIT 2: Dasatinib Superior to Imatinib in Newly Diagnosed CML

Results from a large phase 3 prospective randomized open-label trial, comparing imatinib 400 mg with dasatinib 100 mg daily, were presented by Stephen O'Brien, MD, professor of hematology, Newcastle University Medical School, Newcastle upon Tyne, England.

December 8th 2014

Evaluating Safety, Efficacy, and Outcomes of TKIs in CML

A poster session on the second day of the 56th annual meeting of the American Society of Hematology, held December 6-9, San Francisco, was dedicated to trials evaluating therapeutic options in chronic myelogenous leukemia. Data presented included safety, efficacy, managing comorbidities, and biological differences that drive response to therapy.

December 8th 2014

TKIs and Molecular Response in CML: the EURO-SKI Study

On the second day at the Annual Meeting of the American Society of Hematology 2014, Francois-Xavier Mahon, MD, PhD, Bordeaux Hospital, INSERM 1035, Bordeaux, France, shared the results from the European LeukemiaNet Stop TKI (EURO-SKI) study; the trial used tyrosine kinase inhibitors (TKIs) in CML to define prognostic markers to increase the rate of patients in durable deep molecular response after stopping TKI.

December 2nd 2014

CDC Designates 35 US Hospitals as Ebola Treatment Centers

Laura Joszt, MA

Although there are no current cases of Ebola in the United States, an increased number of hospitals are now equipped to treat patients with the virus while minimizing risk to healthcare workers.

November 30th 2014

US Hospitals Wary of Caring for Ebola Patients Because of Cost and Stigma

US officials trying to set up a network of hospitals in this country to care for Ebola patients are running into reluctance from facilities worried about steep costs, unwanted attention, and the possibility of scaring away other patients.

November 23rd 2014

Officials Revise Goals on Containing Ebola After Signs of Wider Exposure in Mali

The leaders of the United Nations and the World Health Organization expressed renewed alarm on Friday about Ebola's tenacity in Africa and, in particular, its potential to ravage a fourth country, Mali, where they said hundreds of people had been exposed to an infected cleric who died last month.

October 23rd 2014

Dr Karen B. DeSalvo Leaves ONC for Ebola Response Team

Laura Joszt, MA

Effective immediately, Karen B. DeSalvo, MD, MPH, will be stepping down as national coordinator for health information technology to assist HHS in the fight against Ebola.

October 23rd 2014

Hospitals' Struggles to Beat Back Familiar Infections Began Before Ebola Arrived

While Ebola stokes public anxiety, more than 1 in six hospitals-including some top medical centers-are having trouble stamping out less exotic but sometimes deadly infections, federal records show.

October 21st 2014

CDC's New Directive on PPE for Healthcare Workers Treating Ebola Patients

The CDC has released guidelines with detailed information on the type of personal protective equipment and the procedure for donning and removing, for healthcare workers managing Ebola patients.

October 17th 2014

FDA Allows Testing of Ebola Experimental Drug by Chimerix

Brincidofovir is being developed by the company against several different viruses, including smallpox and Ebola.

October 15th 2014

Report of Second Texas Nurse With Ebola Fans Hospital Safety Fears

Mary K. Caffrey

Health officials from CDC and the state of Texas are reeling in the wake of today's report that a second nurse at the Texas hospital that treated Ebola patient Thomas Eric Duncan has tested positive for the virus. The news came while health leaders were still trying to determine how 26-year-old nurse Nina Pham contracted Ebola while caring for the first patient diagnosed with the disease on US soil. Meanwhile, the largest nurses' union says its members report that most hospitals are not ready for an Ebola patient.

October 15th 2014

ICD-10 Would Help US Better Track Ebola

If Congress had not pushed back the deadline for implementing ICD-10 last spring, the United States now would have a specific code for tracking Ebola, according to an infographic from the Coalition for ICD-10.

October 14th 2014

Are EHRs to Blame in the Dallas Ebola Case?

With the enormous amount of data being collected and entered into EHRs, the human brain (in this case the physician brain) is probably tuning out information that would probably stay if there was a conversation with the patient instead.

October 2nd 2014

In Texas Ebola Case, Was Patient's Insurance Status an Issue?

Mary K. Caffrey

As Texas health officials monitor 80 people for symptoms of the Ebola virus, the question arises: could this public health threat have avoided if the ER at Texas Presbyterian admitted the patient with the disease when it first had the chance? Was the patient's insurance status an issue, as some have suggested?

September 8th 2014

Superbugs an Increasing Problem in Hospitals

Laura Joszt, MA

For patients who develop infections while staying in a hospital, the chances of it becoming drug resistant increases 1% for each day of hospitalization, according to researchers from the Medical University of South Carolina (MUSC).

August 25th 2014

DepoVax Technology Proves Effective in NIH-Sponsored Ebola Vaccine Study

The study, conducted in cynomolgus monkeys, explored several candidate vaccines from NIAID's preclinical services program.

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