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…
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…
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…
Want some more news, events and announcements on hemophilia? We got 'em! [one_half] [/one_half] [one_half_last] FDA Clears New Drug for Hemophilia B Treatment Virtual-reality Game Helps This Boy…
Fino a poco tempo fa, il termine ROHHAD non significava nulla. Ma poi ho sentito parlare di un bambino canadese di 11 anni chiamato Denika Bailey che ha questo raro…
L'emiplegia si verifica normalmente durante la gravidanza, o molto rapidamente dopo la nascita. I genitori sono fortunati ad avere alcuni momenti con il loro bambino prima che questa condizione cambia…
L'hémiplégie se produit normalement pendant la grossesse, ou très rapidement après la naissance. Les parents ont de la chance d'avoir quelques instants avec leur enfant avant que cette condition ne…
Je suis sûr que vous avez entendu parler de la SLA après que le «Défi du dégel de glace» ait balayé la nation en 2014, mais peu ont probablement pris…
Sono sicuro che hai sentito parlare di ALS dopo che la "Ice Bucket Challenge" ha spazzato la nazione nel 2014, ma pochi ancora probabilmente hanno preso il tempo per ricercare…
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