The medical community has been aware for some time now that patient involvement in his or her treatment is a critical part of therapeutic development. Biopharmaceutical companies acknowledge their obligation…
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…
Neuroscience News recently carried an article heralding reports of researchers' finding that upwards of twenty percent of lethal glioblastoma brain cancers may be treatable by newly developed drugs. Currently, the…
Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and…
According to a study on hindawi.com, the presence of abnormally elevated levels of serum IgG4 was correlated with a greater likelihood of patients living with autoimmune hepatitis to present with…
Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…
Takeda has just announced that their application to the the FDA for their eosinophilic esophagitis (EoE) investigative therapy has received Priority Review. This therapy is called budesonide oral suspension or…
Ryan Kress was crowned Ms. Wheelchair Virginia 2020 and Camille Schrier was crowned Miss America 2020. Besides their titles, what brings these two individuals together is their medical diagnosis with…
Written by Kendra J. Bjoraker, Ph.D., L.P. Living well with a chronic condition such as Fabry can seem contradictory. Following diagnosis, you don’t have a shelf in your mind that…
In October, I sat down with Laura and Ryan Nitahara to discuss their experience with Krabbe disease, a rare genetic lysosomal storage disorder and progressive neurological condition. Prompted by their…
In late December 2020, biopharmaceutical company AbbVie announced via press release that the prescribing information for IMBRUVICA (ibrutinib) is now updated to include long-term safety and efficacy data for…
On January 10, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared positive data from its Phase 3 GENEr8-1 clinical trial. The clinical trial is evaluating an investigational gene therapy…
In a recent press release from mid-January 2020, the Pulmonary Fibrosis Foundation (PFF) announced that its registry, the PFF Registry, was being sponsored by biotechnology United Therapeutics. This sponsorship…
According to an article in BioSpace, Dyne Therapeutics has recently announced data from preclinical trials of its FORCE platform. This program is aimed at creating treatments for myotonic dystrophy type…
In an exciting press release, gene therapy platform company Lysogene announced that it had received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and Research Ethics Committee. As…
According to a story from GlobeNewswire, a new partnership between the precision genetic medicine company Akouos, Inc. and and Blueprint Genetics has produced the Resonate program, which aims to improve…
Did you know that a common food-borne illness might increase the risk of developing a type of rare brain cancer called glioma? According to Science Daily, a recent study suggests…
Recently, Pulmonary Hypertension News shared some burgeoning medical news: that the DNMT3B gene has now been linked to the development of pulmonary hypertension (PH) and a rare form of chronic high blood…
Waldenstrom macroglobulinemia (WM) is incredibly difficult to treat. In addition to having no cures, the condition only has one approved treatment option. However, researchers now believe that combining drugs, specifically…
As reported in WTNH; in Stafford Springs, CN, the Pokorny family was rudely awakened to the world of rare diseases when their new daughter Olivia was born with spinal muscular…
According to a story from news-medical.net, a team of scientists affiliated with Massachusetts General Hospital has conducted a study revealing that gene therapy could be an effective treatment for tuberous…
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 ANCA Vasculitis News, the FDA recently approved Riabni (rituximab-arrx) for the treatment of adult patients with two specific subtypes of ANCA vasculitis: microscopic polyangiitis (MPA) and granulomatosis with…
In early January 2021, biopharmaceutical company BlueRock Therapeutics ("BlueRock"), in collaboration with the Memorial Sloan Kettering Cancer Center, announced via press release that its Investigational New Drug (IND) application for…
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…
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