As reported on NPR, Tatiana Schlossberg, journalist and granddaughter of former U.S. President John F. Kennedy, has disclosed that she is battling a rare and
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has launched an investigation into reports of serious adverse events, including one fatality, associated
Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking
Editor’s Note: Patient Worthy is pleased to share this article from our friends at Elephants & Tea, originally written by Cecily Liu. To see the
The United Leukodystrophy Foundation (ULF) is proud and excited to announce a partnership with Marc Nolan, a Chicago-based footwear brand, who is launching a new
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib), an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, for adults with neurofibromatosis
Hundreds more patients in England with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) will gain earlier access to glofitamab (Columvi) after new guidance from
Vera Therapeutics has taken a major step toward transforming treatment for immunoglobulin A nephropathy (IgAN), submitting a Biologics License Application to the U.S. Food and
My journey started abruptly last year. It was a completely normal day—until it wasn’t. I’m an RN, and I slept most of the day before
I’m Jenna, a 36-year-old married mother of one son, and I have neuroendocrine cancer (NETs). It is a rare type of cancer, and 60% of
Arrowhead Pharmaceuticals has secured its first U.S. approval with Redemplo, an RNA interference therapy cleared by the FDA for adults with familial chylomicronemia syndrome (FCS).
See additional Important Safety Information below. Please read the accompanying full Prescribing Information, including Boxed WARNING, also available at NUPLAZID.com. You are encouraged to report
As reported by PharmaBiz, the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion supporting Orphan Drug Designation (ODD) for
The FDA has given early approval to Enhertu as a frontline treatment for HER2+ metastatic breast cancer. Read more here:
patientworthy.com/2025/12/30/fda-approves-enhertu-combination-for-first-line-her2-positive-breast...
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GLP-1 Drugs Show Promise in Lowering Epilepsy Risk Among Diabetes Patients bit.ly/3MGhnaf Read now at PatientWorthy.com
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