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The California Institute for Regenerative Medicine (CIRM) has funded a study by a company called Forty Seven, Inc. The clinical trial is investigating the benefits of a drug therapy for two cancers…
Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…
According to a story from mdmag.com, a recent study that was presented at the 2019 meeting of the American Society of Clinical Oncology has demonstrated the potential of a two…
Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…
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The Asco Post recently interviewed Dr. Jean Pierre Bizzari, the Executive Vice President of Celgene, a multinational bioparmaceutical company. The interview covered various aspects of research and development (R&D)…
Patient Worthy's partner the MDS Foundation is hosting a MDS Patient & Family/Caregiver Forum Baltimore, Maryland in conjunction with the University of Maryland Marlene and Stewart Greenebaum Cancer Center. This…
In a world that is just beginning to make space for the open discussion of transgender individual's life experiences, there's still a huge gap in the conversation regarding healthcare. This…
Personalized medicine is the latest craze in patient care. In essence, it refers to the development of treatment options which take into account the patient as an individual. It reduces…
GT Biopharma has just announced that their Phase 1 clinical trial for Mastocytosis, Myelodysplastic Syndrome (MDS), and Acute Myeloid Leukemia (AML) has been authorized to begin. Although this investigation had previously…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. When: Saturday, April 27, 2019 Where: West Penn Hospital 4800 Friendship Avenue Pittsburgh, PA 15224 Wintergarden Conference Center – First…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…
Celgene Corporation has recently announced two exciting updates for the rare disease community! Update #1 The first big announcement from Celgene was that their supplemental New Drug Application (sNDA) for…
The very first patient in Medigene AG's clinical trial examining MDG1011 has officially received the treatment. This is the company's first human clinical trial for this TCR therapy. The trial…
What is the Patient Advocate Foundation? The Patient Advocate Foundation (PAF) is a non-profit, formed in 1994 that has one primary aim- eliminate the barriers to treatment too many patients…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. This forum will take place in Phoenix, Arizona. When: Saturday, March 9th, 2019 Where: Sheraton Crescent…
The government shutdown hasn't had any startling affects on rare disease research yet, but drug developers have voiced their concern that it could soon begin to hinder the research process.…
Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…
According to a story from Science and Technology Research News, a group of researchers from the Cleveland Clinic led by Dr. Aziz Nazha will soon present a new myelodysplastic syndromes…
According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that…
A recent article titled ‘How I use molecular genetic tests to evaluate patients who have or may have myelodysplastic syndromes’ published in the journal Blood discusses the role of genetic testing for…
According to a story from Hematology Times, a recent study found that genetic sequencing after the completion of a hematopoietic stem cell transplant could provide doctors with vital information about…
According to a story from Medpage Today, three recent research studies have managed to reveal new biomarkers for myelodysplastic syndromes. Biomarkers are a crucial factor for understanding a medical condition…
The company Imago Biosciences, which focuses on developing anti-cancer therapies, is carrying out a Phase 1/2a clinical study of an investigational drug called IMG-7289 as a potential treatment for…
A group of researchers studying the causes underlying myelodysplastic syndromes have found that HIF1A may play an important role in these conditions. For more detailed information, you can read the…
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