This post was originally published on this site Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come…
Conditions
Conditions
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
This post was originally published on this site Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health…
Tally of Risk Factors Can Identify Myeloma Patients at Risk of Deep-vein Blood Clots, Study Suggests
This post was originally published on this site A simple score of risk factors could help alert doctors to a likelihood of dangerous blood clots, called venous thromboembolism, forming as a side effect of immunomodulatory therapies (IMiD) for multiple myeloma. Researchers established the set of factors — based on a person’s age, race, and medical…
Gray Foundation Awards $3.75M to Study of BRCA Mutations in Ovarian and Other Cancers
This post was originally published on this site The Gray Foundation awarded a $3.75 million grant to a researcher team to study how BRCA1 or BRCA2 mutations, along with other genetic anomalies, increase the risk of breast, ovarian, and other cancers. The “team science grant” will fund the research for four years. The project is…
Maintaining Friendships Is Challenging for Caregivers and Those with Alzheimer’s
This post was originally published on this site This week people around the world celebrated International Day of Friendship. In 2011, the United Nations General Assembly designated July 30 as a day to recognize the power of friendship to address disharmony in the world by “accumulating bonds of camaraderie and developing strong ties of trust.”…
Chemical Compound Can Restore Neural Function in Familial Alzheimer’s, Study Finds
This post was originally published on this site A mutation in a gene linked to familial Alzheimer’s disease promotes the disease by reducing the points of contact between nerve cells and impairing neuronal communication, a study has found. A chemical compound, called EVP4593, partially rescued the mutation’s negative effects, suggesting it may hold a…
First Generic Version of Navelbine Launched in Europe for Breast, Lung Cancers
This post was originally published on this site Lotus Pharmaceuticals has launched the first generic version of Pierre Fabre’s Navelbine (vinorelbine) in soft capsule form in Europe for the treatment of advanced breast cancer and advanced non-small-cell lung cancer, the company has announced. Vinorelbine soft capsules are now being marketed across Europe, including in France,…
First Patient Dosed in Phase 1 Trial of Antibody for Advanced Multiple Myeloma, Teneobio Announces
This post was originally published on this site A Phase 1 trial evaluating Teneobio’s bispecific antibody TNB-383B as a treatment for multiple myeloma has started dosing patients. This safety and tolerability trial is recruiting people with advanced disease, who received at least three prior lines of therapy. The open-label study (NCT03933735) will first test increasing…
$566K Grant Will Support Research on Use of Contraceptive Pill to Prevent Ovarian Cancer
This post was originally published on this site A $566,000 grant, awarded via the Ovarian Cancer Research Foundation, will support research in Australia that aims to establish the use of contraceptive pills as an effective strategy for preventing ovarian cancer. The three-year grant will enable researchers to continue exploring how changes in female reproductive hormones…
Xtandi Delays Disease Worsening in Metastatic Hormone-sensitive Prostate Cancer, Study Says
This post was originally published on this site Adding Xtandi (enzalutamide) to androgen deprivation therapy (ADT) significantly delays disease progression or death in men with metastatic hormone-sensitive prostate cancer (mHSPC), regardless of how spread the disease is, a Phase 3 clinical trial shows. It also improved several other patient outcomes, extending the time to a new cancer…
‘Off-the-shelf’ CAR T-Cell Therapy Moving Toward 1st Clinical Trial with Takeda
This post was originally published on this site A new chimeric antigen receptor (CAR) T-cell therapy derived from induced pluripotent stem cells (iPSCs) has been transferred to Takeda from the Center for iPS Cell Research and Application (CiRA) at Kyoto University to prepare for clinical testing. Under the terms of the collaboration, Takeda shall retain the…
Phase 1 Trial of Betalutin, a Radioimmunotherapy, in Advanced DLBCL Now Fully Enrolled
This post was originally published on this site The Phase 1 trial evaluating Betalutin — an investigational tumor-targeting antibody labeled with a type of radiation — in people with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) is now fully enrolled, its developer, Nordic Nanovector, announced. The ongoing LYMRIT 37-05 trial (NCT02658968) includes 18 patients who are…