BeiGene has just announced that China has given conditional approval to Pamiparib, a PARP inhibitor used as a therapy for BRCA associated ovarian cancer, fallopian tube cancer, and primary peritoneal…
As COVID-19 makes its way around the globe, scientists are discovering new ways the infection will linger in the months and years to come. As reported in John Hopkins Medicine,…
Dr. Duneel Dhand, an internal medicine physician and author, told KevinMD about two events in his life that motivated him to treat his patients with compassion. A Doctor’s Character As…
Enzyvant has just announced that they have resubmitted their application of RVT-802 to the FDA as a therapy for pediatric congenital athymia. Their first application was submitted in 2019. The…
Diagnosis can be difficult to achieve for rare disease patients. Some patients experience years of searching for the correct diagnosis, with others receiving the wrong one along the way. Changes…
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…
Normally, amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder, affects individuals between ages 50-60, with prevalence increasing with age. Once diagnosed, patients typically survive for another 3-5 years. However,…
According to a recent news release, the Lennox-Gastaut Syndrome (LGS) Foundation shared that its 6th Annual Walk 'n' Wheel event will take place on June 5th, 2021. The event…
On May 24, 2021, biopharmaceutical company NeoImmuneTech, Inc. shared that the first patient was dosed in a pilot study centered around NT-I7 (efineptakin alfa). Altogether, the study is designed…
Pfizer Rare Disease is an approach taken by pharmaceutical giant Pfizer to develop treatments for rare and orphan diseases. In the past, the company has worked with the National Newspaper…
As reported in Biospace, the biotech company Jasper Therapeutics Inc. has announced the start of a Phase 1/2 clinical trial on a potential conditioning agent, JSP191, to facilitate hematopoietic cell transplants,…
In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…
Brigatinib has been approved for the treatment of ALK+ metastatic non-small cell lung cancer (NSCLC), as well as ALK+ NSCLC patients who have received prior treatment with crizotinib. While it…
The diagnostic journey for rare disease patients can be long and frustrating. In fact, 28% of rare disease patients spent a minimum of seven years waiting for their diagnosis, and…
Intravenous immunoglobulin (IVIG) has been used to treat autoimmune conditions for years, and it is now being explored as a treatment for certain neurological diseases. Recent research has investigated IVIG…
Over 50 years ago, levodopa was approved to treat patients with Parkinson's disease. To this day, levodopa, administered via pills or intravenously, remains the standard-of-care. But according to Parkinson's News…
Misophonia, which literally translates to hatred of sound, is a rare condition that causes affected individuals to have severe emotional reactions to common sounds, such as chewing or tapping. For…
A majority of individuals who have strokes are over the age of 60. But what happens when younger individuals have unexplained strokes? What causes these to happen? According to a…
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