According to a recent article in Fierce Biotech, most cancers progress through constant cell division. Scientists at Vanderbilt University are determined to find the reason for this mysterious cell division.…
According to a story from Charcot-Marie-Tooth News, a recent study has demonstrated the potential impacts of an RNA-based therapy as a treatment for type 1A Charcot-Marie-Tooth disease. The treatment was…
In a recent press release, biopharmaceutical company Swedish Orphan Biovitrum AB ("Sobi") shared that the first patient was dosed in a Phase 3 clinical trial evaluating avatrombopag for pediatric patients…
Dicaprio Bootle is on a mission: to become a star athlete in the NFL. However, he's preparing to give back along the way to his goal. Dicaprio is fundraising for…
In March 2021, the FDA expanded access approval to biotechnology company Longeveron Inc.'s investigational Lomecel-B to a pediatric patient with hypoplastic left heart syndrome (HLHS). According to a press…
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…
Together, biopharmaceutical company Sanofi and biotechnology company Regeneron Pharmaceuticals, Inc. ("Regeneron") have been working to develop Libtayo (cemiplimab) for patients with advanced cervical cancer. According to a press release,…
Running a foundation for a rare disease isn’t always an easy thing. I receive emails daily asking me for information about Twin Anemia Polycythemia Sequence, a rare disease of monochorionic twins affecting just…
Newron Pharmaceuticals and Zambon have entered into an agreement that has the potential to produce a label extension for a treatment for Parkinson's. The agreement is focused on safinamide as…
As reported in NewsWise; rare drugs are few and far between, with a cliental of less than 200,000 Americans dissuading drug companies from taking on the enormous charge of developing…
Pamrevlumab is a first-in-class antibody that treats Duchenne muscular dystrophy (DMD) by inhibiting the effects and activity of connective tissue disorder growth factor (CTGF). It is being developed by FibroGen,…
Most people who are unhappy with the price of their medication are not aware of drug prices in the truly high-end segment of the market. GoodRx, the prescription drug watchdog,…
The Patient-Centered Outcomes Research Institute (PCORI) has announced that it will be contributing funding for four new studies focused on four different rare diseases. The funding will top out at…
Since its conception, biotechnology company Chinook Therapeutics, Inc. ("Chinook") has worked to develop therapeutic solutions for patients with kidney diseases. Now, the company enrolled the first patient in a…
During the drug development process, multiple studies are performed to evaluate efficacy, safety, tolerability, pharmacokinetics, and other factors. One of these studies is called a multiple ascending dose study, during which…
In 2015, I published my first book, Diagnosis Dystonia: Navigating the Journey. It is a combination autobiography, self-help, education book that provides treatment options, coping strategies, skills for daily living,…
For nearly 130 years, pharmaceutical company Merck & Co ("Merck" or "MSD") has been working to develop unique therapeutic solutions for those with rare cancers and a variety of…
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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