Currently available therapeutics may not be adequately effective in the fight to treat late-stage acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Researchers and drug developers are working to identify…
A Phase 2 clinical study set out to determine whether prexigebersen, alongside decitabine and venetoclax, would be effective in treating and stopping the progression of a rare cancer of…
For the first year and a half of her life, Audrey seemed to be a healthy child. Her parents Tenneil and Tom had no real concerns about her health. So…
Since 2021, biotechnology company Foghorn Therapeutics ("Foghorn") has been developing its therapeutic candidate FHD-286 for a number of malignancies. In one Phase 1 study, researchers are testing the pharmacokinetics,…
Effective June 1, 2023, the FDA lifted its clinical hold on Foghorn Therapeutics’ Phase 1 study of FHD-286 dose escalation in patients with acute myeloid leukemia and myelodysplastic syndromes. The…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
For patients with refractory or relapsed KMT2A rearrangement or NPM1-mutations, the AUGMENT 101 study of the investigational menin inhibitor revumenib yielded impressive results. According to an article in MedicalXpress,…
The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…
16-year-old Tae’Von Perry has always been incredibly active in his local community: from acting as a worship leader at Encounter Church to singing, playing sports, and dancing with Company D.…
The Orphan Drug Act was passed with a goal in mind: to incentivize the development of therapeutics for orphan drugs. These drugs are used for patients with rare conditions,…
Although kinase-targeting drugs have been used for many years in the fight against AML and other cancers, according to a recent article published in FierceBiotech, scientists at the Cold Spring…
Currently, clinical-stage oncology company Sumitomo Pharma Oncology, Inc. is working to develop DSP-5336 for patients with acute myeloid leukemia (AML). The company even began recruiting for a Phase 1/2 study…
Researchers acknowledge that the primary cause of failure of stem cell transplants is disease relapse. This holds true specifically for allogeneic hematopoietic stem cell transplants where a cancer patient…
In the United States, the Orphan Drug Act was established to incentivize drug developers and other stakeholders to begin developing therapies for rare diseases. Orphan Drug designation is now granted…
Recently diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations now have a new treatment option, as the FDA has granted approval to TIBSOVO (ivosidenib tablets)! This is an expansion…
Lately, the first thing that enters a person’s mind if they encounter an unusual pain is that they may have cancer. Yet that was the last thing Georgina Masson, an…
The FDA has just provided AB001 the Orphan Drug designation for both acute myeloid leukemia (AML) and pancreatic cancer. This small molecule was developed by Vopec Pharmaceuticals and Agastiya Biotech.…
A recent article in Labroots stated that the majority of patients who have preleukemic disorders may not be candidates for leukemia. Estimates are that there are about ten to fifteen…
April 21st is AML World Awareness Day! Acute myeloid leukemia (AML) is a rare cancer which affects people around the world. On awareness day, patients, caregivers, healthcare organizations, advocates, physicians,…
Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. It spreads rapidly. Therefore precise and swift treatment is critical. In a recent interview with Medical Life…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Fast Track designation is granted by the FDA to facilitate the development and expedite the review of drugs to both fill unmet needs and treat serious or life-threatening conditions. Through…
Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…
An article in PubMed dated October 8, 2021 outlines immune therapies treating myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients. Both cancers are hematologic malignancies that begin in the…
The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this designation, "rare" conditions are those affecting…
© Copyright Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
