According to Charcot-Marie-Tooth News, the CMT Research Foundation has announced that they will provide additional funding for the development of a gene therapy for Charcot-Marie-Tooth disease Type 1 (CMT1A). The…
On March 11, 2021, Patient Worthy attended an educational seminar hosted by the Cleveland Clinic titled Neuromyelitis Optica Spectrum Disorder (NMOSD): Educational Seminar for Patients, Family Members and Caregivers Focus…
Genetec Engineering and Biotechnology News recently carried a story giving credit to Baylor College researchers for finding microbes living in the gut that are associated with specific symptoms of neurological…
The French National Agency for Medicines and Health Products Safety, also known as ANSM, has granted Temporary Authorization of Use of berotralstat for those living with hereditary angioedema (HAE) above…
Fast track designation is granted by the FDA. If a drug developer receives this designation, they begin a process to develop and expedite review for drugs or biologics which treat…
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…
CRISPR has seen a number of developments recently; 2020 was a good year for gene therapy. Medical professionals are excited by the improvements made and motivated to make further discoveries.…
In early March 2021, the National Comprehensive Cancer Network (NCCN) shared a new set of NCCN Guidelines for Histiocytosis. Histiocytosis, or hystiocytic disorders, are a group of rare conditions characterized…
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