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…
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…
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.…
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…
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…
In a recent press release, genome editing company Intellia Therapeutics, Inc. ("Intellia") announced its recent MHRA authorization to begin a Phase 1 clinical trial to evaluate its therapeutic candidate NTLA-2001. The…
Dr. Philip Mease at Washington University’s Medical Center likes to tell his patients that they are unique. According to a recent Healio report, Dr. Mease spoke at the 2020 Clinical…
Many patients with rare conditions know that sometimes it can be extremely difficult to find targeted treatment options. In fact, a large number of rare conditions still have no approved…
Telehealth is something that has become common during the coronavirus pandemic. It allows people to see their physicians without stepping out of the home, drastically lowering the risk of exposure.…
According to a story from Charcot-Marie-Tooth News, the drug company DTx Pharma has recently obtained critical funding that will go towards the development of new RNA-based treatments for Charcot-Marie-Tooth disease…
According to a story from BioSpace, SIRION Biotech GmbH and Mustang Bio, Inc. have announced a new agreement for licensing the use of SIRION's proprietary LentiBOOST™ technology for use by…
According to a story from Newswise, it was more than three years ago that Maxford Brown, 16, awoke one morning feeling out of sorts. Maxford, who lives with Down syndrome,…
Many people within the rare disease community share a common experience: feeling isolated or misunderstood because of your condition. In some cases, such as conditions causing chronic pain or fatigue,…
At the very core of genetic diseases are - well, our genes. However, each year, an estimated 400,000 infants are born with de novo mutations, or new and spontaneous gene mutations not…
According to a story from BioSpace, the biopharmaceutical company Kite announced recently that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has released…
According to a recent article in Healio, Therapeutic Options for Interstitial Lung Disease was one of the topics covered at the Congress of Clinical Rheumatology 2020. Dr. Elizabeth Volkmann co-director…
When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…
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