Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…
A recent announcement in Biospace by Capsida Biotherapeutics and CRISPR Therapeutics of their newly-formed partnership offers renewed hope for amyotrophic lateral sclerosis (ALS) and Friedreich’s Ataxia patients. The companies believe…
Impel NeuroPharma has been working on a campaign focused on migraines. It is called Reroute Migraine Relief, and it was built in coordination with an artist who suffers from migraines…
According to a story from nbcnewyork.com, A resident of Sussex County, New Jersey has recently tested positive for a rare infectious disease called Jamestown Canyon virus, or Jamestown Canyon encephalitis.…
A two-month-old baby recently traveled to Dubai to receive treatment at Al Zahra Hospital Dubai (AZHD), according to an article in the Khaleej Times. The child, who is from Oman,…
Strongbridge Biopharma has just announced that the FDA has decided to review the New Drug Application for RECORLEV, a therapy for endogenous Cushing's syndrome. The target action date is January…
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…
According to a study published in PLOS Genetics, a recent genetic study of hepatocellular carcinoma (HCC) cancer tumors has revealed vital information that could one day lead to a new…
During the European Hematology Association (EHA) Annual Congress (EHA2021), which took place virtually this year, hematological experts and stakeholders discussed new advances in the field. According to Fierce Biotech, one…
As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…
In early June 2021, Epidermolysis Bullosa News reported that Filsuvez (Oleogel-S10), a topical gel for epidermolysis bullosa (EB), received Priority Review designation from the FDA. Filsuvez Developed by Amryt Pharma,…
The Rare Disease Legislative Advocates (RDLA) webinar for the month of June took place on June 17, 2021. This month's webinar provided updates on a variety of legislative topics that…
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 a news release from June 23, 2021, Revolo Biotherapeutics ("Revolo") shared that the FDA approved an Investigational New Drug (IND) application for the company's '1104 peptide treatment. Altogether, '1104…
Mark Dalziel has big plans for this weekend; he's going to bike the length of Scotland within two days. He has a very good reason for doing so, which is…
Orphan Drug designation is a status granted to drugs or biologics intended to treat patients with rare conditions, defined as those affecting under 200,000 Americans. According to Pulmonary Hypertension News,…
Mal de débarquement, or "sickness of disembarkment," is a phenomenon which causes a bobbing, rocking, or swaying sensation, alongside other symptoms. With many people, mal de débarquement only lasts…
As reported in Charcot-Marie Tooth News; this year, Eurordis has kicked off a campaign to make sure those with rare diseases don’t get left behind in the coming century. Small…
Until recently, multiple myeloma patients were reliant upon treatment with immunomodulatory drugs, anti-CD38 antibodies, proteasome inhibitors, and autologous (patient-derived) stem cell transplants. According to a recent article in CancerConnect, patients…
Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV), or alveolar capillary dysplasia (ACD) for short, is a rare, congenital, and life-threatening disorder. Without receiving lung transplants in infancy,…
The National Medical Products Administration (NMPA) of China has recently approved surufatinib for drug registration for the treatment of advanced pancreatic neuroendocrine tumors (pNETs). HUTCHMED Limited recently announced the news,…
Transcription factor SOX 11 is encoded by the SOX11 gene. Altogether, this protein is known to play a role in embryonic neurogenesis and tissue formation. However, an estimated 90% of patients with…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
HAE Junior’s drawing exhibition unveiled the dreams, wishes, and ambitions of children & teenagers living with hereditary angioedema (HAE). The patient organization HAE Junior organized a drawing exhibition named We…
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…
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