AstraZeneca, a global leader in pharmaceuticals, has taken a significant step into the rare disease arena in South Africa, aiming to improve diagnosis, treatment, and support for patients facing these…
Sanofi has announced a significant regulatory milestone for its investigational therapy SAR446523, a monoclonal antibody targeting GPRC5D, after the U.S. Food and Drug Administration (FDA) granted it orphan drug designation…
Australian athlete Tiana Death has bravely shared her journey after being struck by autoimmune encephalitis, a rare and serious brain disease that upended her life and forced her to relearn…
The U.S. Food and Drug Administration (FDA) has broadened the approval of Empaveli (pegcetacoplan), a complement inhibitor, to include the treatment of two rare and serious kidney diseases: C3 glomerulopathy…
In a significant advancement for cancer treatment, the U.S. Food and Drug Administration (FDA) has granted accelerated approval to Bavencio (avelumab) for the treatment of patients with locally advanced or…
In Chapel Hill, North Carolina, a family’s life changed dramatically when their young twin daughters were diagnosed with Batten disease, a rare and devastating genetic disorder. According to MSN.com, the…
Biogen has achieved a significant regulatory milestone with the UK Medicines and Healthcare products Regulatory Agency (MHRA) granting approval for Qalsody (tofersen), a treatment specifically developed for a rare and…
Lancet Oncology Recently published reports updating the findings from the phase 3 DREAMM 7 trial involving 494 patients averaging ages 64.5 years with the median follow-up of 39.4 months. Accordingly, patients…
Sydney health authorities are responding to a rare disease outbreak after a confirmed case of melioidosis resulted in one death. The incident, reported by MSN.com, has brought the little-known but…
A growing body of evidence suggests that GLP-1 receptor agonists, a class of medications commonly used to treat diabetes and obesity, may play an important role in managing hidradenitis suppurativa…
Novel radioactive medicine may be an inconvenient read for many people, but if you or someone you care about has metastatic breast cancer the words will soon become part of…
Kevan Chandler, was born with a rare, and progressive neuromuscular disease: spinal muscular atrophy (SMA). At age 33, he needs assistance with almost all activities of daily living, from eating…
A recent study has unveiled a novel treatment that could significantly lessen brain damage following a stroke, offering hope for more effective therapies in the future. According to World Pharma…
Inflammatory bowel disease (IBD), which includes Crohn’s disease and ulcerative colitis, is a lifelong condition that disrupts the gastrointestinal tract and significantly impacts quality of life. While IBD has no…
Calidi Biotherapeutics has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting Fast Track designation to its experimental therapy, CLD-201 SuperNova, for the treatment of glioblastoma.…
How do you prioritize your responsibility as a caregiver when there is illness in the family? Especially when your husband is facing new rounds of chemotherapy and a fourteen-year-old autistic…
A recent study has revealed encouraging results for mirikizumab, an investigational monoclonal antibody, in addressing one of the most distressing symptoms of Crohn’s disease: bowel urgency. According to Medscape, this…
Each year, nearly half a million Americans are affected by Lyme disease, a tick-borne illness that, if untreated, can lead to serious complications like arthritis, heart issues, and neurological disorders.…
AbbVie, a global biopharmaceutical leader, has reported disappointing results from a phase 2 clinical trial evaluating its interleukin-1 (IL-1) targeting drug, ABBV-668, as a monotherapy for patients with moderate to…
A new Missouri law is shining a spotlight on the vital need for awareness of rare childhood diseases, thanks in large part to the advocacy efforts of a Eureka, Missouri…
Acknowledgement: This poem, originally written by Olivia Thompson, was provided to Patient Worthy by our friends at Elephants & Tea. To see the original, please click here. One welcomes a…
Microbiotica, a UK-based biotechnology company focused on developing microbiome-based therapeutics, has reached a significant milestone by completing patient recruitment for its phase 1b clinical trial in ulcerative colitis (UC). According…
A recent study highlighted by World Pharma News reveals that maintaining a healthy diet can significantly slow the progression of chronic diseases among older adults. Chronic diseases—such as diabetes, heart…
Acknowledgement: This article was originally written by Gillian Sapia, a mother of a child living with galactosemia, for Underground Rare Road. To see the original article, please click here. When…
In our second installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over the next four weeks. …
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