According to a story from The Print, Neha Shoriwala, a mother from New Delhi, is urgently seeking help for her son Harshil, who lives with Wilson's disease, a rare illness…
When drugs enter into clinical studies, both patients and drug developers alike hope for success. These therapies could transform the treatment sphere, especially in disease states with limited or…
Historically, drug development within the rare disease space has been stifled; companies tend to focus on developing therapies for bigger patient populations that will be more impactful and more…
The FDA has granted IDEAYA Biosciences fast track status for IDE161 as a treatment for HR+, Her2- breast cancer. The therapy is a selective inhibitor of ADP ribose glycohydrolase…
CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…
According to a story from Pharmaceutical Business Review, the UK's National Institute for Health and Care Excellence (NICE) is backing daridorexant (marketed as QUVIVIQ) as a treatment for chronic insomnia…
Patients with severe asthma with an eosinophilic phenotype who are 12 years of age or older may now receive treatment with FASENRA as an add-on maintenance treatment. Note that…
J.D. Martinez is a designated hitter and outfielder for the LA Dodgers. According to a story from The Santa Clarita Valley Signal, he also lives with asthma, which can have…
Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
Every three to four minutes, someone in the U.S. is diagnosed with a blood disease such as lymphoma, leukemia, or sickle cell anemia. As reported in the Pittsburgh Post-Gazette,…
Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…
In 2010, Eddie Vedder (well known for his musical career in Pearl Jam) co-founded the EB Research Partnership (EBRP) alongside wife Jill and a group of parents whose children…
In 2005, the U.S. Food and Drug Administration (FDA) approved abatacept for the treatment of moderate-to-severe rheumatoid arthritis. Twelve years later, in 2017, the FDA expanded the drug's approval…
Arcus Biosciences and Gilead Sciences issued a press release announcing encouraging overall response rates and six-month progression-free survival rate for their combination treatment of domvanalimab plus zimberelimab and chemotherapy…
In the past, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for different forms of cancer, such as non-small cell lung cancer (NSCLC) and melanoma. More recently,…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Imagine the frustration of Faustina Cavero, unable to follow her maternal instincts that told her something was wrong with her baby. Doctors at two different hospitals disagreed with her and…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
On October 24, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
Only around 350 to 500 people in the United States are diagnosed with Creutzfeldt-Jakob disease (CJD) each year. Unfortunately, this disease often causes rapid cognitive decline and physical deterioration,…
According to a story from Healio, 21 people living with the disease prurigo nodularis took part in a qualitative study over the phone in which they shared the ways in…
Currently, there are no FDA-approved treatments designed for cognitive impairment related to Huntington’s disease. However, to improve the lives of those affected, additional research and therapeutic development are urgently…
The two trials evaluating lacutamab, an investigational therapy developed to treat T-cell lymphomas, were put on partial hold by the FDA. The patient died of hemophagocytic lymphohistiocytosis, which is…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
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