Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company based in Utah, has announced that its investigational drug, ofirnoflast (HT-6184), has received Orphan Drug Designation (ODD) from the U.S. Food and Drug…
In a week of significant developments for the biopharma industry, an FDA advisory committee issued pivotal recommendations on two high-profile blood disorder drugs, while PureTech Health announced a leadership change…
This article is a continuation of a previous story, and is an excerpt from original author Isabella Cristobal's post. To read the first part of the article, please click here.…
In 2020, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to magrolimab for the treatment of newly diagnosed myelodysplastic syndromes (MDS). This designation is designed to expedite…
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…
The 1st International Workshop on Myelodysplastic Syndromes was held this year in Miami, FL from June 24-26. This in-person event was a convergence of researchers and clinicians to discuss advances…
Karyopharm Therapeutics, a pharmaceutical company focusing on novel cancer therapies, has issued a statement via PR Newswire that the FDA has granted regulatory designations to eltanexor, Karyopharm’s Selective Inhibitor…
In an August 3rd, 2022 news release from Syros Pharmaceuticals (“Syros”), the company shared that its product tamibarotene had received a positive opinion for its Orphan Drug designation application.…
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…
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…
Orphan drug designation is granted to drugs or biologics intending to treat, diagnose, or prevent rare diseases or conditions. Within the United States, a rare condition is one affecting under…
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…
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…
Each year, approximately 1 in every 1 million infants is diagnosed with pediatric myelodysplastic syndromes (MDS), a group of rare disorders characterized by the abnormal or deficient formation of blood…
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 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…
Over the past few weeks, Centenary College has partnered with Be The Match to host a stem cell donor drive. The first two drives were on November 18th and 19th,…
ABC News recently featured an article about Isabella Cristobal and Sergio Soto. They decided to get married shortly after learning that Sergio’s cancer had spread and he had only…
The outcome of myelodysplastic syndrome (MDS) varies. The disease can lie dormant for decades, or it can become fatal in a matter of months. According to a recent article in…
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…
Science Magazine recently joined other news sources in reporting that Bluebird Bio’s Phase 3 clinical trial investigating a treatment for a neurological disease was put on hold by…
In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…
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…
© 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.
