Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
Toxicity resulting from cancer therapy is usually the result of the repeated administration of cancer drugs. On the other hand, the efficacy of CRISPR-Cas9 has been largely affected by problematic…
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CRISPR-Cas9’s Gene Editing Potential in Cancer Therapy
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A recent announcement by Gilead Company citing data from Kite Pharma, one of its premier companies, set out remarkable results from Kite’s Zuma-1 clinical trial. The findings confirmed that…
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Infusion of Yescarta Resulted in 44 Percent 4-year Overall Survival Rate in Large B-cell Lymphoma
Most people are aware of various symptoms caused by the stress that we experience on a day-to-day basis. However, there are a variety of physiological reactions that affect our peripheral…
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Exposure to Stress Leaves an Imprint on the Brain Leading to Stress-Related Insomnia
Stanford University Geneticist Ronald Davis faced one of the most challenging research projects of his career. According to a recent article in The Scientist, ten years ago Whitney, his son,…
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A Father’s Mission to Discover a Cure For His Son’s Chronic Fatigue Syndrome
According to a recent report in Cancer Network, the results from CARTITUDE-1 were well received. CARTITUDE-1 is a phase 1b/2 clinical trial of the CAR T-cell treatment, Cilta-Cel, for the…
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Impressive Results for Cilta-Cel to Treat Patients With Relapsed/Refractory Multiple Myeloma
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A recent article in the Encinitas Advocate heralded the dedication of Stan Crooke, n-Lorem Foundation’s CEO and founder. The nonprofit Foundation’s team acts as middlemen, working with patients who…
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The N-Lorem Foundation is Developing Treatments for Patients With Ultra-Rare Diseases
Kura Oncology, a San Diego biopharmaceutical company, was recently featured in a Globe Newswire article, announcing its first-in-human dose escalation of KO-539 being evaluated in the Komet-001 trial (NCT04067336). The…
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Kura Oncology Announces Clinical Data for KO-539, an AML Treatment
According to a recent article in NewsWise, researchers have discovered that families with the PCSK9 gene remain healthy through their eighties and nineties. The study has been published in the…
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Several Canadian Families With the PCSK9 Gene Mutation May Have Found the Fountain of Youth
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…
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A CRISPR Tool With the Potential to Halt the Progression of Sickle Cell and Beta Thalassemia
The New England Journal of Medicine recently carried a report citing data from a Phase 3 clinical trial involving 307 people diagnosed with MSI-H-dMMR metastatic colorectal cancer. MSI-H (high levels…
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Pembrolizumab’s Targeting of MSI-H-dMMR Colorectal Cancers More Effective Than Chemotherapy
ASH 2020: CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope Published: Dec. 7, 2020 BioSpace CRISPR Therapeutics and Vertex Pharmaceuticals presented new data on…
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CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope
The United States is coping with an obesity epidemic. It is therefore critical to understand the distribution of body fat and its effect on treatment. A recent article in Healio…
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Study: The Relationship Between Obesity, Lung Diseases, and Pulmonary Hypertension in Transplant Patients
Xavier Alford told BBC News how he vividly recalls the day, twelve years ago, when he was told by his doctor that he had a very rare disease. His doctor…
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Filmmaker Produces a Documentary That Helped Him Cope With His Paralysis From Guillain Barre Syndrome
A webinar hosted by CureDuchenne featuring an informative program by Mesentech is scheduled for 4:30pm EST on Thursday, December 10th. The community is invited to join in and learn directly…
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CureDuchenne Announces Webinar Featuring New Approach To Bone Regeneration in DMD
Axcella is a biotechnology company that has developed a new system of treating complex diseases. Its lead candidates are products that treat two liver diseases, as well as NASH, which…
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Axcella Presents its Investigational Products to Treat Liver Diseases and Type 2 Diabetes
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Mesentech and CureDuchenne have announced a partnership that includes generous funding from the Charles H. Hood Foundation. The partnership has developed a targeted therapy for reversing bone wastage in boys…
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Joint Funding Partnership Announced to Promote Potential Bone Growth Therapy for DMD
WCVB NewsCenter 5 recently published a news item featuring Nancy and Paul Burke and their three daughters. The Burke family has been living in the shadows of the deadly Sanfilippo…
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The Burkes Are Racing the Clock to Help Save the Lives of Their Three Daughters With Sanfilippo Syndrome
An article recently appeared in Health News by NPR describing progeria, an extremely rare disease. There are only twenty people in the United States diagnosed with progeria. The disease causes…
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A Drug that Extends the Life of Children With Progeria Has Been Approved by the FDA
Sorreto Therapeutics recently announced through Globe Newswire that it has filed an application for its investigational new drug COVI-DROPS (STI-2099). The study will investigate the safety and efficacy of the…
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COVI-DROPS Enter Human Study to Fight Mild Cases of COVID-19
COVID-19’s tentacles have invaded the New Drug Applications (NDAs) for 2020. A recent article in Fierce Pharma reported that the FDA has been especially vigilant in scrutinizing NDAs this year,…
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The FDA Found Issue With Several NDAs in 2020, Partly Due to the COVID-19 Pandemic
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The latest news from Israeli scientists, published in the Times of Israel, is that researchers at Tel Aviv University have accurately targeted cancerous cells in mice using CRISPR.…
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Tel Aviv University Team Uses CRISPR Technique to Destroy Metastatic Cancer Cells in Mice
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According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
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Brainstorm Shares the Results of its Phase 3 Study of NurOwn to Treat ALS
A recent article in BioSpace highlighted presentations by Twist Bioscience, a rapidly growing genomic and synthetic biology company. Twist presented its product line at the virtual 2020 Annual Meeting of…
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Twist Bioscience Gives Impressive Presentation at the Virtual American Society for Human Genetics 2020 Annual Conference
Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…
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Two Clinical Trials for POMC and LEPR Deficiency Obesity Led to a Significant Reduction in Body Weight and Hunger
According to an article in Biospace, CureVac of Boston, MA, with headquarters in Tübingen, Germany, just announced interim results from its Phase 1 study evaluating CVnCoV, a vaccine candidate against…
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CureVac Joins the Race to Develop a Vaccine Against COVID-19 With Its Successful Phase 1 Trial