Lee Neal age 38 and his wife Jessica age 35 were on holiday when they noticed a slight swelling in their toddler’s eye. Sixteen months later, their baby died. Now…
Recent advances within the medical field have prompted the development of RNA therapeutics for many different applications. These therapeutics have the potential to greatly change the treatment landscape and create…
According to a story from Kalimpong News, over 300 children with rare diseases in India are still waiting for the release of pledged funds from the Union Health Ministry so…
According to a news release from biopharmaceutical company BridgeBio Pharma, Inc., the first patient was dosed in a Phase 1 study. Researchers are evaluating the safety, tolerability, pharmacodynamics, and pharmacokinetics…
Ryner Lai, MBBS, a medical graduate of Queen Mary, London University, is a physician and writer with a passion to use medical research as a way of improving patients’…
According to a story from Market Screener, the clinical stage biopharmaceutical company Kringle Pharma, Inc., has recently announced an update on the progress of its phase 2 clinical trial. The…
Karyopharm Therapeutics, a pharmaceutical company focusing on novel cancer therapies, has issued a statement via PR Newswire that the FDA has granted regulatory designations to eltanexor, Karyopharm’s Selective Inhibitor…
According to a story from Cancer Network, the treatment landscape for myelofibrosis, a cancer of the bone marrow, still has substantial unmet need. An important component of treatment are JAK…
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 story from Medical Xpress, a medicine used to treat high blood pressure and enlarged prostate could be a potential therapy for amyotrophic lateral sclerosis (ALS), a rare…
On August 10, 2022, KTVU anchor Leslie Griffith passed away in Lake Chapala, Mexico, where she had been living for the past six years. Leslie was 66 years old…
According to a video posted on Targeted Oncology, adverse events are a significant concern for people that are receiving treatment for the chronic form of graft-versus-host disease. In the video,…
Near the start of August 2022, biotechnology company Inmagene Biopharmaceuticals ("Inmagene") and biopharmaceutical company HUTCHMED shared via news release that the first participant had been dosed in a Phase…
As part of its 2022 webinar series, the Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP) hosted a webinar on August 17, 2022 titled "Addressing the Gaps in Clinical…
According to a story from Ankylosing Spondylitis News, Health Canada has recently approved upadacitinib (marketed as Rinvoq) as a treatment for people living with ankylosing spondylitis. The medicine is specifically…
Both the 3rd Annual Gene Therapy for Neurological Disorders meeting and the ADVANCE 2022 Sanfilippo Community Conference were held in July 2022. During both meetings, researchers presented new data from…
Don't forget to check out Part 1, where we discussed what multiple sclerosis (MS) is, went over Mo's diagnostic journey, and began talking about the importance of awareness. Treating Multiple Sclerosis…
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…
Six years after receiving a lung transplant a young woman 21 years of age noticed an article in Reforestemos Patagonia © about another young woman who was waiting for a…
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…
In the United States, Orphan Drug designation is a special status granted to drugs or biologics intending to treat, prevent, or diagnose a rare disease or condition. A "rare" condition…
For as long as she can remember, 30-year-old Ankona “Mo” Das has loved traveling. Traveling has provided a way to break out of her shell, to experience new landscapes, and…
For those living with sickle cell disease (SCD), treatment and management can sometimes be difficult. Currently, there is no cure for SCD. Patients may manage their condition using antibiotics,…
Patient Worthy would like to wish a Happy 2nd Birthday to one of our partners, the TAPS Support Foundation. It is hard to believe that it has only been two…
Don't forget to read Part 1 of our interview, where we discussed what Costello syndrome is, Ryan's son Reynolds' diagnosis, and what spurred the family to want to make a change. mejo…
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