Happy Friday! November 1 is International Acromegaly Day. For those unaware, Acromegaly is a rare condition characterized by the presence of an elevated level of growth hormone and typically effects…
According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…
Last week, from October 22nd through the 25th, marked the American Society of Nephrology (ASN) Kidney Week. According to a press release, Synthetic Biotic medicine developer Synlogic, Inc. ("Synlogic") presented…
2326 words (source) vs. 503 words (mine) - 6% match Near the start of October, global biopharmaceutical company Jazz Pharmaceuticals ("Jazz") announced positive safety and efficacy data from their Phase…
Recently, specialty pharmaceutical company Xeris Pharmaceuticals ("Xeris") announced that its injectable treatment XP-0863 received Fast Track designation for the treatment of patients with acute repetitive seizures. Xeris created this treatment…
Early on October 28, 2020, Insmed Inc. ("Insmed") announced that it received Marketing Authorization from the European Commission for ARIKAYCE. This therapy is designed for adult patients with nontuberculous mycobacterial…
Written by Rebekah Palmer We have passed the 30th anniversary of the Americans with Disabilities Act. The landmark legislation that prohibits discrimination of disabled people in public spaces is rarely…
Colton’s Story Debbie Moore was 18 months old when she was diagnosed with X-linked hypophosphatemia (XLH) a rare, deforming, and painful bone disorder. XLH causes softening of the bones…
A recent article in Medical Express reminded readers that our DNA goes back many generations, yet it was possible for a genome archeologist to call upon ancient elements that are…
Novartis has recently released interim data on iptacopan, according to a press release. The data on the C3 glomerulopathy treatment was released at the American Society of Nephrology 2020 Annual…
Dr. David Pisetsky, a translational researcher at the Medical Center of Duke University, recently described his team’s new approach to treating potential Lupus patients. The doctor told the Rheumatologist of…
The documentary "Walk in My Shoes” takes viewers through a lifetime with cystinosis with Chandler and his family. After Chandler was diagnosed with the rare disease as an infant, making…
According to a story from SMA News Today, the Patient Access Network (PAN) Foundation announced that it is now offering financial assistance to help patients living with the rare disease…
In a recent study published in Clinical and Experimental Immunology, researchers determined that patients with high ANCA antibody levels are at an increased risk of having ANCA-associated vasculitis (AAV) relapses. According…
A new study has uncovered that using a hypomethylating agent in combination with a BCL2 inhibitor called Venetoclax may be advantageous as a therapeutic option for individuals with acute myeloid…
The FDA has officially approved remdesivir for the treatment of adults and children (over the age of 12 and weighing at least 40 kg) who have been hospitalized for COVID-19.…
Researchers at the Mayo Clinic have collaborated with the international community to develop a potential way to test for spinocerebellar ataxia type 3 (SCA3), also called Machado-Joseph Disease. Additionally, they…
A recent study published in PLOS ONE has reinforced the importance of vaccinations specifically for those already fighting a rare disease. This study was written by Sylvie Lesage from the…
Many different approaches have been offered by scientists to stem the overwhelming tide of Alzheimer’s disease (AD). Fifty percent of primary care physicians believe that the medical profession is not…
As the medical field develops, more companies are looking to create innovative ways to address cancer treatments. In the case of global pharmaceutical company Daiichi Sankyo Company, Ltd. ("Daiichi Sankyo"),…
Monitoring tumors is an essential part of cancer treatment; it allows doctors to make informed decisions regarding which therapies to use. For many forms of cancer, medical professionals have been…
Twins Iris and Clarisa Valdez recently shared their story of courage and hope and thanked the City of Hope care team for its guidance and support. But most importantly…
As reported in BioSpace, the FDA shocked Mesoblast executives, a stem-cell research company, when this October, they rejected the Biologics License Application (BLA) for the drug Ryoncil, meant to treat…
A new treatment for Duchenne muscular dystrophy (DMD) has recently been approved by the FDA. The therapy is called viltolarsen and it was created by Nippon-Shinyaku and the National Center…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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