According to a recent article, professional athletes have been forced into early retirement due to arrhythmias. Arrhythmias, which are potentially fatal, affect those who exercise too much at a higher…
At first, Dr. Rebecca Elon began to notice her husband’s personality changes and an occasional lapse in judgment. These incidents occurred in 2013. Although Dr. Elon’s husband, William Adler III…
Recently, Kezar Life Sciences Inc. ("Kezar") released interim data and preliminary results from part two of the Phase 2 MISSION clinical trial. According to BioPharma Journal, the results highlight the…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
In the National Football League's My Cause My Cleats campaign, NFL players are able to show their passions beyond the game and represent a cause that is important to them through customized…
According to a recent article, being able to detect cancer as soon as possible is vitally important as it allows time to administer curative treatments. Scientists are hopeful new treatments…
Nick Cannon has announced that he will be taking some time off to be with family and mourn after the death of his son, Zen. The sad news was broken…
According to a recent article, a little boy’s parents are bringing awareness to the rare genetic disorder called uncombable hair syndrome after he was diagnosed at just 14 months old.…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
On December 9, 2021, the National Organization for Rare Disorders (NORD) hosted a webinar program titled "Health Equity and Rare Disorders." This webinar was the first of a three-part series…
Novartis has recently announced the results of their Phase II BYLieve trial, which is investigating Piqray® (alpelisib) as a treatment for hormone-receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-)…
Meniere's disease is a rare inner ear disorder characterized by "attacks" of tinnitus, ear pain, ear pressure, and dizziness. While doctors have long pondered the exact cause of this condition,…
Dr. Deborah M. Stephens from the Huntsman Cancer Institute recently presented at the 39th Annual Chemotherapy Foundation Symposium Innovative Cancer Therapy for Tomorrow (CFS). The presentation surrounded how critical identifying…
According to a recent article, a Vietnam veteran is spreading awareness about the struggles of accessibility for treatments for his rare genetic disease alpha-1 antitrypsin deficiency (A1AD). Alpha-1 Antitrypsin Deficiency…
In a recent news release, biopharmaceutical companies AbbVie, Inc. and SpringWorks Therapeutics, Inc. ("SpringWorks") shared that the two had entered into a joint clinical trial collaboration agreement. Through this collaboration,…
On December 10, 2021, biotechnology company Active Biotech shared that the first subject was dosed within a Phase 1 clinical trial evaluating laquinimod for non-infectious non-anterior uveitis. What is particularly…
In a recent news release, biopharmaceutical company Aligos Therapeutics, Inc. ("Aligos") shared that the first healthy volunteers were dosed in a Phase 1 clinical trial evaluating ALG-055009 for nonalcoholic steatohepatitis.…
On December 6, 2021, biotechnology company Longeveron Inc. ("Longeveron") announced that its investigational therapeutic candidate Lomecel-B earned Orphan Drug designation from the FDA. The therapy is designed to treat patients…
According to Pharmacy Times, the FDA recently granted Priority Review status to luspatercept-aamt (Reblozyl). The therapy, for which its developer has submitted a supplemental biologics license application (sBLA), is designed…
In 2016, Marley Berthold was born. But doctors, and her parents, noticed some concerning signs. Marley's silver hair had fallen out in clumps; she had macrocephaly (an abnormally large head)…
Nancy Yu co-founded AllStripes with a vision in mind: to unlock more treatment options for those with rare diseases. In Part 1 of our interview, Patient Worthy sat down with…
Nobody wants to get sick on vacation; it can ruin a time meant to be fun and relaxing. Unfortunately, Kai McCulloch, a 13-year-old from Scotland, experienced this firsthand. They were…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
A recent article in Science Daily reports that researchers at the Copenhagen and Lund Universities, using animal models, killed bacterial meningitis without the use of antibiotics. Estimates from the WHO…
Krystal Biotech has just announced results from their Phase 3 trial called GEM-3, and the results were all positive! This trial investigated bermemagene geperpavec (B-VEC), or VYJUVEKTM as a potential therapy…
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