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…
Positive results have been announced from a Phase 2 trial of olutasidenib, an investigational selective mIDH1 inhibitor used in combination with azacitidine for the treatment of the mIDH1 form of…
The FDA grants the Orphan Drug designation to treatments indicated for rare diseases, which are defined as affecting less than 200,000 people in the U.S. This designation is meant to…
Dr. Richard Stone recently interviewed with Targeted Oncology to discuss challenging acute myeloid leukemia subtypes and a list of available therapies. Dr. Stone was asked to look back at…
Breast cancer is a result of a cell in the breast mutating and multiplying rapidly. There are over 275,000 occurrences of breast cancer in the U.S. each year. However, new…
According to a recent article in MedicalXpress, researchers led by professor of medicine Dr. Jing Chen uncovered new data showing how acute myeloid leukemia (AML) becomes active and progresses. The…
In mid-August 2021, biotechnology and pharmaceutical development company Biosight Ltd. shared that it had begun a Phase 2 clinical trial regarding one of its proprietary treatments - and that the…
In the Phase 3 PANTHER clinical trial, also known as the Pevonedistat-3001 study, researchers were evaluating pevonedistat in conjunction with azacitidine for patients with acute myeloid leukemia (AML), myelodysplastic syndromes…
When Yang Siyang learned his eight-year-old Lillian has the rare childhood cancer acute myeloid leukemia, he felt overwhelmed by shock, grief, uncertainty. There are many intense and complicated emotions that…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
In the past, researchers have shown that acute myeloid leukemia (AML) may be driven by certain genetic mutations. For example, some mutations might cause the abnormal or excess production of…
In a recent company update, via AccessWire, biotechnology company Aptevo Therapeutics Inc. ("Aptevo") shared positive results from a Phase 1 dose-escalation trial. During the trial, Aptevo evaluated APVO436 for patients…
In a recent press release, immuno-oncology and biopharmaceutical company GT Biopharma, Inc. ("GT") shared the release of new data and results from the Phase 1/2 GTB-3550 TriKE clinical trial. Through…
Cancer Network recently reported that Jazz Pharmaceuticals’ Vyxeos was approved by the FDA to treat therapy-related pediatric patients one year or older who were newly diagnosed with acute myeloid…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to BioSpace, Kronos Bio will continue with its plans for a Phase 3 trial of entospletinib after a positive meeting with the FDA. One of the meeting's focuses was…
As reported in NewsWise, as rare diseases get their time under the microscope, growing evidence uncovers medical distinctions. New lines are drawn, delineating new disorders to be defined and classified.…
The first patient has been dosed in a Phase 1 clinical trial, which is exploring Curis Inc.'s CA-4948 in conjunction with ibrutinib for hematologic malignancies. Examples of hematologic malignancies include…
In an early February press release, pharmaceutical company Astellas Pharma Inc. ("Astellas") shared that XOSPATA (gilteritinib) was given conditional approval in China for the treatment of patients with FLT3-mutated…
Poly (ADP-Ribose) polymerase inhibitors (PARP inhibitors) are used to treat neoplasms. They are frequently used as a therapy for ovarian cancer. A recent study has attempted to detect the possibility…
Dr. Andrew Brunner, an oncologist at Massachusetts Cancer Center was recently interviewed by Targeted Oncology. The discussion centered around a phase 1b clinical trial (NCT03066648) investigating sabatolimab together with hypomethylating…
While novel treatments have been rapidly emerging onto the drug scene, new treatment options for pancreatic cancer have fallen behind. But now, according to OncLive’s interview with Dr. P.A. Philip,…
© Copyright 2024 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.
