Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…
Extremely positive results have just been announced from a 5-year follow-up study of a systemic juvenile idiopathic arthritis (sJIA) treatment called Kineret. This treatment has been approved for use in…
According to a story from globenewswire.com, the biopharmaceutical company Strongbridge Biopharma plc, recently presnted a new analysis of data from the company's Phase 3 trial. This trial was testing the…
Farber Disease Farber Disease is a rare condition otherwise known as Acid Ceramidase Deficiency. It is a lysosomal storage disease caused by a missing ceramidase enzyme. This is due to…
According to a story from BioPortfolio, the biopharmaceutical company AlzProtect recently announced that PAREXEL Biotech, which is a subsidiary of PAREXEL International Corporation, will be expected to conduct a Phase…
According to a story from finanzen.ch, the drug companies Pfizer and Merck recently released a joint statement in which they announced that the companies' Phase III clinical trial would be…
Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…
Cystic Fibrosis Cystic Fibrosis (CF) is a rare disease caused by a defected CFTR gene. It results in a buildup of mucus which impairs breathing and makes the lungs susceptible…
Alex Barker never planned on marrying someone with his same rare diagnosis, but he has to admit, the shared experience has made him and his wife closer. Alex has Moebius…
Pattern.Org Pattern.org was created by the Rare Cancer Research Foundation, a nonprofit dedicated to improving the lives of rare cancer patients by accelerating research. Pattern.org specifically, is a branch of…
The most common treatment for autoimmune conditions such as ankylosing spondylitis, plaque psoriasis, Crohn's disease, juvenile idiopathic arthritis, and rheumatoid arthritis is currently a drug called Humira, created by AbbVie.…
"Something's just not right - this is beyond coincidence." Ocular Melanoma is a rare cancer which only affects 5 out of every one million people. Typically, it affects older white…
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Following a record number of approvals in 2018, the FDA has recently approved two new drugs with many more anticipated approvals later in the year. Trazimera to Treat Human Epidermal…
The longer patients live without a diagnosis, the longer they are living without the proper care and the proper treatment. The problem is twofold when a patient is diagnosed incorrectly.…
According to a story from Financial Buzz, the biopharmaceutical company Albireo Pharma, Inc. issued a statement on Rare Disease Day which emphasized the company's continued commitment to progressive familial intrahepatic…
The Patient-Centered Outcomes Research (PCORI) The PCORI is a nonprofit whose primary main mission is to help support research that will allow patients to make informed healthcare decisions. What makes…
Cassie Bebout is an Auburn University undergraduate student, on track to graduate this May. She chose the school for one primary reason- it is the home of Dr. Doug Martin…
Happy Wednesday Everyone! We hope everyone had a good St Patrick's Day. Today, we're highlighting a story from PW contributor Denise Crompton on grief. This is followed by an article…
Shifting Contracts Over the years the United States healthcare system has shifted more toward reimbursement models that are outcome-based instead of service-based. In essence, this means that instead of paying…
Rett Syndrome Rett syndrome is a rare and progressive neurodevelopmental disorder caused by mutations to the MECP2 gene. While it affects all ethnic and racial groups, it is found almost…
According to a story from prnewswire.com, the biopharmaceutical company Dompé recently announced that the company has received the 2019 Industry Innovation Award from the National Organization of Rare Disorders (NORD).…
Progeria and Atherosclerosis Progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS), is a rare disease affecting just 400 people across the world. It is caused by a mutated LMNA gene.…
According to a story from finanznachrichten.de, biotechnology companies are starting to set their sights on treating deadly diseases that are lacking effective treatment options, including some diseases that are considered…
Phase 1 Trial A Phase 1 trial by Gamida Cell is examining a new potential treatment option for patients with multiple myeloma and relapsed/refractory non-Hodgkin's lymphoma who have not responded…
According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…
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