According to a story from Medscape, in a recent study the drug capivasertib appeared to be effective as a treatment for certain metastatic cancer tumors. Classified as an inhibitor of…
According to a story from Globe Newswire, the genetic medicine company Sarepta Therapeutics has announced top-line data from part one of its clinical trial. This trial is investigating its experimental…
The statistics are grim. A survey of 12,000 US nursing homes finds over 56,000 COVID related deaths as of July 2020. This number represents ten percent of all cases in…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
BPD Bronchopulmonary dysplasia (BPD) is a rare condition that primarily affects the lungs of premature babies (though it can affect full term newborns as well). Most children with BPD are born…
Dr. Cory M. Resnick, Oral & Maxillofacial Surgeon at Boston Children’s Hospital and Assistant Professor at Harvard Medical School, describes the exciting progress he has made on the Prenatal Diagnosis of Pierre Robin Sequence. Interview by Philippe Pakter, chairman of Pierre Robin Europe.
According to BioSpace, recent research has demonstrated that CRISPR gene editing could be the answer to treating progeria, or Hutchinson-Gilford progeria syndrome. The Broad Institute and NIH have collaborated to…
As reported in EMJ Reviews; the novel coronavirus has gripped the world— fueling a wave of medical innovation and prioritization. However, the excess of need has made the normal flow…
According to EMJ, findings from an analysis spanning 20 years has found that weight loss surgery used to address obesity also significantly lowers a person's risk of developing pancreatic cancer.…
Immunoglobulin A nephropathy (IgAN) is a glomerular disease which can lead to kidney failure. Between 70 and 100 percent of all IgAN patients also are diagnosed with microscopic hematuria, but…
According to a story from Charcot-Marie-Tooth News, a recent cell-based study has revealed that Charcot-Marie-Tooth disease type 1C and type 4J share a common pathway malfunction. This is despite the…
Research During COVID Many medical research projects have faced hurdles since the pandemic has started. Regulators have changed development timelines, and many have had to delay their clinical trials due…
The FDA has recently cleared an Investigational New Drug (IND) application for FBX-101, allowing it to move into a Phase 1/2 clinical trial. This gene therapy was developed for the…
Many people have questions about the COVID-19 vaccine, a number of them pertaining to the speed at which it was developed and approved. Rare disease patients are no exceptions. Barbra…
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…
Eurordis The European Organization for Rare Diseases (Eurordis) is a nonprofit organization which organizes over 930 rare disease groups into one alliance. Groups have joined the network from 73 different countries.…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
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…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is a rare disease that causes progressive muscle loss and resultant muscle weakness. It is caused by a lack of motor neurons. It…
According to a story from Targeted Oncology, Dr. Bradley Monk talked about the importance of molecular testing in determining the treatment course for ovarian cancer at the Targeted Oncology Case…
Landos Biopharma has recently released data from their Phase 2 trial of BT-11, a treatment for ulcerative colitis. The results were positive, making this therapy a possible viable treatment for…
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,…
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…
Blueprint Medicines Corporation has just announced that they have submitted a supplemental New Drug Application (NDA) to the FDA. This application is for a therapy called AYVAKIT, which was developed…
A study published in BMC Pulmonary Medicine focuses on the role of endothelial progenitor cells and fibrocytes in lung sarcoidosis. They specifically investigated how these cells, along with CD34 + cells, spurred…
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