Rare Disease

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

By Giuliana Grossi

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

By Giuliana Grossi

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

By AJMC Contributor

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

By Karen Jacobson-Sive

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

By Karen Jacobson-Sive

October 28th 2024

More News

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.

August 16th 2014

Ebola - Underscoring the Global Disparities in Health Care Resources

August 14th 2014

FDA Issues Warning on Fraudulent Ebola Prevention And Treatment Products

The FDA, in a press release, has warned consumers to be aware of such products being sold online.

August 12th 2014

Affordable Care Act Projected to Drive Up Prescription Drug Spending in Coming Years

Specialty drugs expected to account for 40% of total prescription spending by 2016, according to Catamaran Corporation.

August 11th 2014

PALB2 Gene May Increase Women's Risk of Breast Cancer

Katie Sullivan, MA

New research found that the risk of developing breast cancer increased in women who had mutations of a gene related to BRCA2, the PALB2 gene.

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.

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.

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.

July 3rd 2014

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

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