According to a story from globenewswire.com, the gene therapy company Sio Gene Therapies, Inc., has recently released encouraging interim findings from its phase 1/2 clinical trial. This trial is evaluating…
Eureka Alert recently published a statement by a team of researchers that early results of a new method of treating Alzheimer’s have been “absolutely spectacular.” The new antibody-based therapy…
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…
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…
According to a recent article, Connie Lee Batlevi, MD, PhD, who is a medical oncologist, recently shared her experiences and knowledge pertaining to the treatment of follicular lymphoma. Follicular Lymphoma…
In June 2021, Health Canada approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF), with at least one F508del mutation, aged 12+. According to a news release from late…
A recent article in Medical Life Sciences alerts physicians to be aware of a deadly skin cancer. It is called cytotoxic cutaneous T-cell lymphoma (CTCL). According to an article in…
A new approach to evaluating the value and efficacy of treatment was developed by associate professor Meredith Regan ScD of Harvard’s Medical School and Dana Farber Institute. A recent article…
CONTENT WARNING: Severe skin wounds and damage Working in rare disease is always hard- It means working in suffering and grief, but even with all our experience, we had to…
According to a recent article from the Irish Medical Times, a combination of surgery and platinum-based chemotherapy is the best treatment for patients with advanced ovarian cancer. Ovarian Cancer Ovarian…
Patient Worthy had the privilege to attend in-person the Periodic Paralysis Conference, which took place from October 30-31, 2021 and was organized by the Periodic Paralysis Association (PPA). This event…
According to a recent article, a recent study from the U.K. showed that emergency methods of managing hyperkalemia, such as the use of insulin/dextrose, are not the most effective treatments.…
When you are looking for new treatments that will either stop or slow the development of Alzheimer’s disease, where would you look? According to a recent article in Gladstone…
CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…
According to a recent article, PharmaTher Holdings Ltd. announces the FDA granted orphan drug designation (ODD) for ketamine in the treatment of complex regional pain syndrome (CRPS). Complex Regional…
According to a recent article from Medscape, Takeda Pharmaceuticals is offering a rebate for brigatinib (Alunbrig) which is used for the treatment of non-small cell lung cancer. Non-Small Cell Lung…
According to a recent article in Biospace, Eisai Pharmaceutical and Biogen Biotechnology recently announced updates to their Phase IIB study 201 and a five-year open-label extension (OLE) studies using lecanemab…
It is always exciting to see the onset of a new clinical trial designed to research therapies to fill unmet needs for patients. According to a news release from biotechnology…
According to a recent article, the overall survival of patients with untreated metastatic non-small cell lung cancer (NSCLC) were significantly improved by the addition of two immunotherapeutic drugs to…
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…
Access Opportunities for Rare Disease Patients At present, there are around 7,000 identified rare diseases, with only 5% having treatments. That leaves more than 95% of rare disease patients with…
A recent article in the journal Nature elaborated on the work of Dr. Ted Love who left early retirement in 2013 to devote his talents and experience to the development…
The National Organization for Rare Disorders (NORD) held its virtual Rare Diseases and Orphan Products Breakthrough Summit on October 18-19, 2021. Patient Worthy was able to sit in on some…
In a news release from September 30, 2021, biopharmaceutical company Arrowhead Pharmaceuticals ("Arrowhead") shared that it had initiated its Phase 2b AROAPOC3-2002 clinical trial and that the first patient…
Dr. Ed Neilan, who is the Chief Medical and Scientific Officer of the National Organization for Rare Disorders (NORD), recently commented that there are approximately more than seven thousand…
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