Many sisters may play pranks or bicker with each other often. According to ABC 11 News, Twelve-year-old Natalia and fourteen-year-old Nyasia Yarrell are no different. One minute they are mad at…
You're happily tucking your toddler in for her afternoon nap when she starts to panic. Where's her favorite blankie and stuffed penguin? A peaceful nap won't occur without it! With…
At any time of the day, in any hospital, a nurse is giving instructions to a parent, spouse, child, sibling or friend of a patient, as to how to take…
Xynomic Pharma, a United States-based oncology research and development company, announced at the end of last month that it had entered into a partnership with the University of California San…
The line between genius and madness is often closer than people realize. Don’t tell Dr. Sergio Canavero that he can’t help his patient with spinal muscular atrophy (SMA). How many…
Communication in the medical field doesn’t happen as much as it should. But, when it does, great discoveries often occur. The thing is, science is complicated. Researchers have so much…
At 5 years old, Holton Jones was diagnosed with ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. According to the Pensacola News Journal, his parents knew something…
The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease. Niemann-Pick disease is a…
In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy held an essay contest for tickets to a Charcot-Marie-Tooth disease event. We asked members of the Charcot-Marie Tooth (CMT) community to tell…
Support for Ofev® just keeps coming! Ofev® (nintedanib) was first approved by the U.S. FDA in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF) after clinical trials, especially the INPULSIS…
Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the risk of multiple myeloma progression or…
A new public cord blood donation center recently opened up in New York. This is the second of this type of facility to open in the state. In the past,…
Adyn’s Dream is a charity in Athens, Ohio. The organization provides live events that benefit families and their children that have received a diagnosis of spinal muscular atrophy (SMA). The…
In Toledo, Ohio an 8-year-old child is one of 100,000 Americans who is affected by sickle cell anemia. According to WNDU, Terrilonnae Chatman has sickle cell anemia, which can cause red…
Due to a rare disease affecting many parts of her body, my oldest daughter, Kelley, saw a number of different doctors in a number of different locations. I clearly remember…
On May 23, 2017, a biopharmaceutical company, ChemoCentryx, Inc., made a huge announcement. Not only has the US Food and Drug Administration (FDA) granted orphan drug designation for their drug, avacopan,…
Elizabeth couldn’t speak. She wasn’t expected to ever utter a word. She couldn’t read. She struggled to make trips outside of the house. Certain smells and loud sounds bothered her.…
GW Pharmaceuticals sta scoprendo ... Epidiolex® (fabbricato e testato da GW Pharmaceuticals) è un candidato medico cannabinoide medico per il trattamento di sindromi di epilessia rara, resistente e precoce, come…
GW Pharmaceuticals découvre ... Epidiolex® (fabriqué et testé par GW Pharmaceuticals) est un candidat cannabinoïde médical leader pour le traitement de syndromes d'épilepsie rares, résistants et débutants tels que le…
De grandes nouvelles pour les patients atteints d'un cancer du sein métastatique muté par le BRCA: un traitement connu contre le cancer de l'ovaire a passé des essais cliniques de…
Aerie Pharmaceuticals announced exciting efficacy results from the second part of its Phase 3 clinical trial of Roclatan at the end of last month. Roclatan is devised for a group…
Notizie grandi per i pazienti con tumore metastatico mutato BRCA mutato: un trattamento con cancro ovarico conosciuto ha superato le sperimentazioni cliniche di fase III per il tumore metastatico mutato…
Alexander Davis è di Tipton, Indiana. Ha vissuto con atrofia muscolare spinale (SMA). SMA lo impedisce di muoversi molto e non è in grado di camminare. Pertanto, ha bisogno di…
Alexander Davis est originaire de Tipton, Indiana. Il a vécu avec une atrophie musculaire spinale (SMA). SMA l'empêche de bouger beaucoup et il est incapable de marcher. Par conséquent, il…
Notizie emozionanti per le persone che vivono con sarcoidosi! Vanderbilt University Medical Center (VUMC) ha sviluppato un Centro Sarcoidosi di Eccellenza. Attualmente, il dottor Wonder Drake è il direttore del…
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