The Marfan Foundation has a mission to improve the quality of life for patients living with Marfan syndrome and other aortic and vascular conditions. This month, in honor of Marfan Syndrome…
A recent study has demonstrated that neuromyelitis optica spectrum disorder (NMSOD) patients treated with eculizumab have a lower risk of adjudicated relapse compared to those treated with placebo. This study…
A new Phase 2 clinical trial will begin this year to explore NKT2152 (a HIF2a-inhibitor) in combination with Fotivda/Tivozanib (a VEGFR tyrosine kinase inhibitor) as a treatment for relapsed/refractory clear…
Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…
A Phase 3 clinical trial for advanced biliary tract cancer (BTC) has found that there is an improved overall survival rate when patients are given the immunotherapy durvalumab in combination…
AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…
PTC Therapeutics Inc. has just announced that their supplemental new drug application for Evrysdi (risdiplam) has been given priority review by the FDA. The aim is to expand the indication…
Jaguar Health and Napo EU S.p.A have just announced that crofelemer, an investigative therapy for short bowel syndrome (SBS) has been granted Orphan Drug Designation by the EMA. Thanks to…
A recent study published in Advances in Rheumatology has uncovered 2 potential biomarkers which could be used to predict which lupus nephritis (LN) patients are at the highest risk of severe renal…
A study recently published in the American Journal of Transplantation evaluated whether or not transplanting the kidney of a pig could be a viable and safe options for human patients. This study…
Tim Smith is the President of Many Faces of Moebius Syndrome (MFOMS), a nonprofit spreading awareness of the rare condition Moebius syndrome. This condition is characterized by the underdevelopment of…
The World Health Organization has recently declared that they will beat meningitis by 2030 by assisting countries in mitigating its spread and minimizing the effects of the disease itself. They…
Moebius Syndrome Awareness Day is January 24th! This event was created in 2011 by the Many Faces of Moebius Syndrome (MFOMS). It is now celebrated across the world, as the…
The Pitt Hopkins Family Conference & Scientific Symposium (PHRF Conference) will be held June 23rd through June 25th, 2022 in Chicago to raise awareness and share research updates for Pitt…
PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
The largest study of benign adrenal tumors has just been completed. Results have been published in the Annals of Internal Medicine. Specifically, this study examined the effect of mild autonomous cortisol…
Saniona has just announced that their Phase 2b clinical trial examining Tesomet as a treatment for Prader-Willi syndrome (PWS) will be beginning soon. This treatment is a combination therapy which…
Three organizations focused on amytrophic lateral sclerosis (ALS) as well as other rare neurodegenerative diseases, have just celebrated the House of Representatives' passage of the ACT for ALS (H.R.3537/S.1813). The…
Researchers from both Kumamoto University in Japan and the Imperial College in London have recently completed a research study in adult T-cell leukemia/lymphoma (ATL), a rare type of leukemia. They…
Kyleigh Kista, a 3 year old from Benonin South Africa needs heart surgery within the next 3 months to save her life. Kyleigh was diagnosed with tetralogy of Fallot with…
X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…
Prometheus Biosciences Inc. has just announced positive results from their Phase 1 investigation for a new treatment for systemic sclerosis-associated interstitial lung disease (SSC-ILD). This trial investigated PRA023, an anti-TL1A…
The Myrovlytis Trust, a UK Charity focused on promoting research and advancing education for rare diseases, and Patient Worthy are now partners! This organization has focused primarily on Birt-Hogg-Dubé syndrome…
Discerna Pharmaceuticals has just announced the results from a Phase 1 trial investigating their GalXC™ RNAi therapy (Belcesiran) for alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The results demonstrated both…
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