3-year-old Ruby Ann Grimes is bubbly, happy, and loving. But each and every day, Ruby faces an ongoing battle: Opsoclonus-Myoclonus syndrome (OMS). Last January (2020), Ruby was first diagnosed with…
According to a November 3, 2021 news release from biopharmaceutical company OS Therapies, the company’s therapeutic candidate OST-HER2 (OST31-164 / Listeria monocytogenes) received Rare Pediatric Disease Designation from the FDA.…
According to a recent article, a study looking at an investigational BTK degrader proved to have important results in patients with chronic lymphocytic leukemia. Chronic Lymphocytic Leukemia (CLL) Chronic lymphocytic…
In late October 2021, the National Institute for Care and Excellence (NICE) in England recommended Givlaari (givosiran) to treat patients with acute porphyria -- or, more specifically, to treat patients…
In 2019, Mike Smith, a father of two, was initially diagnosed with ocular melanoma in his right eye. Shortly after, Mike made the necessary - but difficult - decision to…
November is officially recognized as National Family Caregivers Month, a time dedicated to recognizing and celebrating caregivers. The entire month is full of recognizing the effort and needs of caregivers,…
According to a recent article, a study looked at the symptoms of Bardet-Biedl syndrome that can affect a patient’s sleep patterns. Bardet-Biedl Syndrome Bardet-Biedl syndrome is a rare disorder that…
According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome. This designation is reserved for…
Casey Martin is well known as the golfer who sued the PGA in 2001 and won a landmark 7-2 Supreme Court decision. According to a report by ESPN, with the…
The FDA grants certain designations, such as the Orphan Drug (ODD) and Fast Track designations (FTD), to medications intended to treat rare and severe disorders. Recently, they granted both of…
According to an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals ("Ionis"), topline data from a Phase 2 clinical trial highlighted the benefits of cimdelirsen (IONIS-GHR-LRx) for patients…
“The patient network is incredibly important,” Lina Williamson, PhD, explained to me. You see, in her childhood, Lina experienced a number of seemingly unexplainable symptoms: severe and sometimes debilitating leg…
In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…
It is often difficult to estimate the prevalence of a rare disease within the United States. For one, it can be difficult to find all of the patients with a…
My story starts with me being a normal, healthy kid. Or was I? My parents would’ve said so, and so would I, I think. I just wasn’t very strong or…
According to a recent article, children with juvenile idiopathic arthritis experienced higher rates of joint inflammation during the COVID-19 lockdown. Juvenile Idiopathic Arthritis (JIA) Juvenile idiopathic arthritis (JIA) proves that…
According to a recent article, patients diagnosed with a rare disease are faced with incredibly high health care costs compared to those without a rare disease. Impact of Rare Disease…
When it comes to advances in hereditary ATTR amyloidosis (hATTR amyloidosis), biopharmaceutical company Alnylam Pharmaceuticals, Inc. ("Alnylam") has been on top of its game. Alnylam helped develop Onpattro and is…
On October 30-31, 2021, the Periodic Paralysis Association (PPA) hosted the Periodic Paralysis Conference in Orlando, FL. This event was an opportunity for patients to meet one another, share experiences,…
LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…
Data from the TELEPRO-II study of Xermelo were recently published in the Journal of Cancer Management and Research. It investigated this oral therapy as a treatment for carcinoid syndrome (CS)…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
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…
According to a recent article from MedPage Today, new clinical guidelines involving parathyroidism were previewed at the American Society for Bone and Mineral Research (ASBMR) meeting last month. Hypoparathyroidism Hypoparathyroidism…
Like every other body part, bones are living tissues and go through several rejuvenation cycles throughout life. Older bone tissues are replaced with newly formed bone tissues in a process…
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