According to CureToday.com, those who have been diagnosed with chronic lymphocytic leukemia, CLL, are now living longer than ever before. This feat can be attributed to the influx of…
Friday September 7th is World Duchenne Muscular Dystrophy Day! Duchenne muscular dystrophy (DMD) is one of nine forms of muscular dystrophy. Patients with DMD cannot make dystrophin in their muscles causing…
According to a story from hms.harvard.edu, a recent study is revealing new information about the connection between aging and neurodegenerative disorders, such as Parkinson's disease, dementia, and amyotrophic lateral sclerosis.…
For comedy and Saturday Night Live fans, Pete Davidson needs no introduction. For those out of the loop, you might now be hearing more of this young comedian who is…
A recent study published in the journal Nature Communications highlights a potential therapy for the treatment of muscular dytrophies and other diseases linked to mutations of the FKRP gene. In the study, the…
According to a story from globnewswire.com, the gene therapy company Adverum Biotechnologies recently announced that the US Food and Drug Administration (FDA) has granted them Orphan Drug designation for their…
According to Digital Health Age, a wearable eye technology may be revolutionary in the lives of those who have Locked-In Syndrome, which is often associated with ALS and other motor…
The investigational drug AT132 has been awarded Regenerative Medicine Advanced Therapy designation for the treatment of X-linked Myotubular Myopathy by the United States FDA. You can read the source press…
Unknown origins of birth. Overcoming enormous personal obstacles at an early age. A mysterious medical diagnosis. Making a drastic new change in life. This could - and is! - a…
I came into the hospital about four days ago at 6 am in extreme abdominal pain. I remember writhing on the emergency room bed and breathing so frantically that my…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…
Living with PKD has been a constant struggle for me but for you to better understand my story, I’m going to start from the beginning when I was first diagnosed.…
Senator John McCain passed away on Saturday, August 25th after his year-long battle with glioblastoma, a rare form of brain cancer. He was four days short of his 82nd birthday.…
Happy Back 2 School Time! As the week comes to a close, we want to spotlight four articles. We have an honest piece from PW contributors Denise Crompton and Tom…
According to a story from BioSpace, Martin Pharmaceuticals' Livantra appears to be on something of a roll. Earlier in the year, the drug received Orphan Drug designation for the treating…
Just this morning, the family of American hero and political icon John McCain announced via a statement he will cease treatment for the aggressive and rare form of brain cancer,…
No one likes to be judged. However, during the many years that I've been involved with rare diseases families, I've learned that some people are very quick to judge parents…
According to a story from thesun.co.uk, Jade Baldwin, 35 had been dealing with mysterious itchiness for years before she sought medical attention for it. While her legs were itchy, there…
According to a story from Newswise, a team of scientists from the St. Jude's Children's Research Hospital have been hard a work conducting an in-depth analysis of a pediatric solid…
This article contains a summary of the first episode of Afflicted A new series on Netflix called Afflicted explores chronic illness. It follows seven people as they talk about their…
According to a report by Newswise, a new form of gene therapy has been developed which may slow or prevent retinitis pigmentosa. Researchers funded by the National Eye Institute (NEI)…
According to the Tampa Bay Times, Aaron McQ did not want to die. He knew, however, with his disease he would inevitably have to. The 50-year-old man from Seattle…
According to The Times of Israel, a group of researchers believe they have found the origin of what could lead to a treatment for amyotrophic lateral sclerosis. Given the debilitating effect…
According to a story by The Japan Times, Masatane Muto relies on a power wheelchair to move around. He is hardly in control of his own body and requires help…
The Alagille Syndrome Alliance has launched its first ever awareness ribbon in support of the Alagille syndrome (ALGS) community! The multi-colored awareness ribbon is meant to represent the complex and uniquely…
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