The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
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…
EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia. As recently reported in the Business Insider, EDIT-301 has just been granted the…
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
Many drugs, whether they are indicated for common or rare diseases, come at very high prices. Zynteglo, an investigational gene therapy for beta thalassemia, is most likely to stick to…
Bristol Myers Squibb (BMS) has been developing luspatercept-aamt - marketed as Reblozyl - as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license…
A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…
According to a recent article, a new stem-cell-based gene therapy is showing promising results for treating beta (β)-thalassemia in a phase 1 clinical trial. Beta Thalassemia Beta thalassemia is a…
According to a recent article, a study shows that treatment with gene therapy reduces the need for patients with non-β0/ β0 genotype Beta-thalassemia (β-thalassemia)to get transfusions. Beta-thalassemia (β-thalassemia) Beta thalassemia…
Clinical trials can be extremely beneficial in learning more about various diseases, as well as the safety, efficacy, and tolerability of potential treatments. According to a news release from…
According to Pharmacy Times, the FDA recently granted Priority Review status to luspatercept-aamt (Reblozyl). The therapy, for which its developer has submitted a supplemental biologics license application (sBLA), is designed…
CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…
As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…
According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
CRISPR has seen a number of developments recently; 2020 was a good year for gene therapy. Medical professionals are excited by the improvements made and motivated to make further discoveries.…
The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…
According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…
According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…
Recently, clinical-stage biopharmaceutical company Imara Inc. ("Imara") announced that the first patient was dosed in the Phase 2b Forte clinical trial. The trial is evaluating IMR-687, a PDE9-inhibitor, for…
According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…
For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…
Recently, Canada made strides towards better, more accessible treatments for patients with beta thalassemia when they approved REBLOZYL (luspatercept). Biopharmaceutical companies Bristol Myers Squibb Canada ("BMS Canada") and Acceleron Pharma…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
Happy Friday! Sept. 24 is FH Awareness Day. Familial hypercholesterolemia is a gentic disorder characterized by the inability of the body to remove LDL -- sometimes called "bad cholesterol" --…
Josephine Bila was born with transfusion dependent beta thalassemia, a rare blood disorder. Recently, Josephine uploaded a video to Youtube in which she talks about her life as a patient,…
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