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…
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…
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,…
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…
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…
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,…
According to a recent press release from Globe Newswire, Albireo Pharma announced that Lancet Gastroenterology published results of the PEDFIC 1 trial evaluating Bylvay® in the treatment of progressive…
This is Part Two of a two part story. Click here to read Part One. Joyce Sanchez, M.D., professor at Wisconsin’s Medical College, supported the women by stating that…
Currently, there are an estimated 30 million people in the European Union (EU) who are living with a rare disease. Drug developers are incentivized to create therapies for those…
This is Part One of the two part story. Stay tuned for Part Two Imagine being told the pain you are feeling throughout your body is not real and then…
In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
According to a story from Market Watch, the drug companies Merch and Eisai recently announced an update on their phase 3 clinical trial. This clinical trial was evaluating a combination…
On July 27, 2022, the National Organization for Rare Disorders (NORD) hosted an online webinar titled "Drug Development for Rare Diseases: A Community Conversation." This program was designed to help…
According to a news release from biotechnology company Kira Pharmaceuticals, its therapy KP104 earned Orphan Drug designation in the United States. KP104 is in development as a therapeutic option…
Results of a recent study published in Medscape Medical News assessed the effect of treatment with radiotherapy on patients who have prostate cancer that is localized. Statistics show that…
According to a story from Myasthenia Gravis News, the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recommended ravulizumab (marketed as Ultomiris) to be approved as…
Researchers acknowledge that the primary cause of failure of stem cell transplants is disease relapse. This holds true specifically for allogeneic hematopoietic stem cell transplants where a cancer patient…
According to a story from Rett Syndrome News, the pharmaceutical company Acadia Pharmaceuticals is seeking U.S. Food and Drug Administration (FDA) approval for its investigational therapy trofinetide. It has been…
2157 words (source) vs. 423 words (mine) - 5% match In the Phase 2 LILAC clinical trial, researchers sought to evaluate the safety, efficacy, and tolerability of litifilimab (BIIB059) for…
At the end of July 2022, biotechnology company Biogen Inc. shared via news release some exciting updates in regards to its therapy tofersen, an investigational treatment for patients with…
Recently a woman was admitted to Odisha hospital where she was successfully treated for her rare blood disorder aplastic anemia. Aplastic Anemia Aplastic anemia is a rare, but serious,…
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