Written by Lori Lawter, MPH We’ve heard a lot about bringing drugs to market lately, especially in light of the COVID-19 vaccine. You might wonder, “Is there a ‘secret sauce’…
According to a recent article, the FDA has given approval for Apellis Pharmaceutical’s Inc.’s Empaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria Paroxysmal nocturnal hemoglobinuria (PNH)…
In a Case-Based Roundtable for Targeted Oncology, Dr. Haris Ali, an associate professor with the City of Hope Department of Hematology and Hematopoietic Stem Cell Transplantation, discussed a case of…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
On September 30, 2021, the National Organization for Rare Disorders (NORD) hosted an educational webinar titled "Rare Cancers: Breaking Down Barriers to Diagnosis, Treatment, and Research." The webinar focused on…
According to a recent press release from Horizon Therapeutics, UPLINZA, the only FDA-approved treatment for AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), may be a viable treatment option for African…
Kristin woke up the morning of her 19th birthday with a startling realization: she was unable to speak. It was hard to describe the feeling, but when she tried to…
Science Direct recently published a Respiratory Medicine Case Report that may impact future Cystic Fibrosis (CF) treatment. The case involves adult brothers herein referred to for purposes of this…
Clinical trials have many endpoints - or milestones they are looking to achieve. These endpoints help signify the safety, efficacy, and tolerability of the treatments. In the Phase 3…
According to a recent article in the International Business Times, a twenty-two-month-old girl living in Odisha, India was diagnosed with pulmonary alveolar proteinosis. The disease causes respiration difficulties due to…
There are inequities when it comes to race in the medical sphere, just as there are in every other facet of life. One specific issue pertains to clinical trials, as…
According to a story from news.cision.com, the US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for CS1, an investigational drug being developed by the…
According to a press release from Business Wire, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive opinion in regards to the…
According to a recent article published in The Online Trading Mentor, Inventiva S.A. (Inventiva) has recently opened enrollment for its Phase 3 trial of Lanifibranor, an investigational treatment for nonalcoholic…
According to a story from ANCA Vasculitis News, the biopharmaceutical company ChemoCentryx has recently made an amendment to its approval application for its investigational therapy. This therapy, called avacopan, is…
There are now plans in place to study a gene therapy for GNE myopathy (GNEM), with human dosing expected to begin in 24 months. The plans were developed by the…
According to a story from Targeted Oncology, the US Food and Drug Administration has given Orphan Drug designation to ARX788, an experimental therapy. The designation was given for the treatment…
Researchers at MD Anderson’s Cancer Center recently announced the results of its Phase II clinical trial. The researchers found that the combination of bevacizumab and atezolizumab decreased clinical trial…
According to a recent article at Pharmacy Times, Infigratinib has been granted accelerated approval for the treatment of those with metastatic cholangiocarcinoma. The treatment is for adults whose cholangiocarcinoma is…
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) has recently approved a new treatment for late-onset Pompe disease. The drug, developed by the biopharmaceutical…
The Arizona Bioindustry Association recently published an article stating that researchers at Arizona University are now proceeding to a Phase 2b clinical trial for a newly developed therapy for Alzheimer's. The…
Breast cancer is a result of a cell in the breast mutating and multiplying rapidly. There are over 275,000 occurrences of breast cancer in the U.S. each year. However, new…
It’s no secret that physical fitness can, in many cases, improve our overall health and wellness. But could being physically fit also improve healthcare and treatment response? According to Medical…
On September 2, 2021, Patient Worthy attended a webinar from the SYNGAP Research Fund titled "Treatments in Development for Epilepsy Syndromes: Opportunities for SYNGAP1." This program provided an overview of…
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