Life has a way of catching us off guard when we least expect it. For me, it all started with severe stomach pains that led to an unexpected hospital visit.…
Research into rare diseases has long faced obstacles, from limited patient numbers to the challenge of replicating disease biology in the lab. A new wave of innovation, highlighted in the…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
Editor's Note: Patient Worthy is honored to share this article from our friends at Courageous Parents Network. Parents of well children regularly receive anticipatory guidance–proactive communication about what’s coming in…
Editor's Note: This article is an opinion piece shared with Patient Worthy from our partner network. Opinion pieces may not reflect the views of Patient Worthy, its employees, or any…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
Families and researchers in North Carolina are making urgent appeals to the FDA, highlighting the desperate need for faster approval of drugs for rare and ultra-rare diseases that threaten children’s…
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 four weeks. This week…
The U.S. Food and Drug Administration (FDA) is reviewing the first medication specifically developed for Barth syndrome, a rare and life-threatening genetic disorder. As reported by STAT, this medication could…
A recent report from NDTV World brings attention to a rare but significant public health event: an American man has tested positive for plague, the infectious disease historically known for…
Health authorities in Queensland, Australia, have issued a public warning after the discovery of Naegleria fowleri—a rare but deadly “brain-eating” amoeba—in a town’s water supply. As reported by The Medical…
A routine evening became pivotal for a Portsmouth family when a bright white reflection appeared in nine-month-old Nora’s left eye under bathroom lights. As was reported by Mirror, what seemed…
Acknowledgement: Patient Worthy is honored to share this story by Erika Stariha, Co-Founder and President of SATB2 Europe. This is Urban, my teenage firstborn. He is joyful. Curious. Full of…
In our third installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
After more than a decade of development, families say time is running out for children with Barth syndrome as the experimental therapy elamipretide faces yet another regulatory delay. Reported by…
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 four weeks. This week…
Millions of social media posts and billions of views have turned TikTok into an influential platform for mental health conversations, but experts warn that using the app to self-diagnose can…
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…
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…
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.…
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…
“I have to be dying”, I said earnestly to my husband for the second time that year, “at the very least, I don’t think there is much more pain I…
A recent research article in ScienceDirect.com details a breakthrough in visualizing the rapid, complex movements of proteins using high-speed atomic force microscopy (HS-AFM). This cutting-edge imaging technology allows scientists to…
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