Paul Williamson has always been a fiercely proud father and an advocate for the rare disease community. His son James was born with Smith-Magenis syndrome (SMS), a rare genetic developmental…
In a Case-Based Roundtable for Targeted Oncology, Dr. Haris Ali, an associate professor with the City of Hope Department of Hematology and Hematopoietic Stem Cell Transplantation, discussed a case of…
For many patients, symptoms of Huntington’s disease appear within midlife: between the 30s and 40s. This progressive disorder causes brain cell death, leading to a loss of physical and mental…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, identify, or diagnose rare or life-threatening conditions. "Rare" conditions are those affecting under 200,000…
Data has recently been announced from a study investigating the efficacy of a new therapy called Alpelisib, for the treatment of PIK3CA-related overgrowth spectrum (PROS). The study was called EPIK-P1.…
Japan's Ministry of Health, Labour, and Welfare has just announced its approval of Alofisel, a stem cell therapy created by Takeda. This approval is specifically for patients who are diagnosed…
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant,…
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
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