Evrysdi (risdiplam) Shows Promising Results in Tiniest Spinal Muscular Atrophy Patients
source: pixabay.com

Evrysdi (risdiplam) Shows Promising Results in Tiniest Spinal Muscular Atrophy Patients

by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…

Continue Reading Evrysdi (risdiplam) Shows Promising Results in Tiniest Spinal Muscular Atrophy Patients
Pantethine Studied for Efficacy in Treating Pantothenate Kinase-Associated Neurodegeneration in Children
source: pixabay.com

Pantethine Studied for Efficacy in Treating Pantothenate Kinase-Associated Neurodegeneration in Children

by Lauren Taylor from In The Cloud Copy Pantothenate kinase-associated neurodegeneration or PKAN is a rare, inherited condition in which the affected child experiences progressive degeneration in certain parts of…

Continue Reading Pantethine Studied for Efficacy in Treating Pantothenate Kinase-Associated Neurodegeneration in Children

Press Release: Bulgarian Cystic Fibrosis Patient Joins International Campaign Standing up for the Rights of Patients to Access Essential Medicines

Bulgarian cystic fibrosis patient joins international campaign standing up for the rights of patients to access essential medicines Rositsa Malinova, a 22-year-old resident of Pazardzhik, Bulgaria, is taking on the…

Continue Reading Press Release: Bulgarian Cystic Fibrosis Patient Joins International Campaign Standing up for the Rights of Patients to Access Essential Medicines
HAE Junior’s Survey Highlights Differences and Similarities of HAE Adult vs. Pediatric Patient Priorities
source: pixabay.com

HAE Junior’s Survey Highlights Differences and Similarities of HAE Adult vs. Pediatric Patient Priorities

HAE Junior is a Czech-based patient organization aiming to improve the quality of life of children & youth with hereditary angioedema (HAE). It was set up in December 2019 in…

Continue Reading HAE Junior’s Survey Highlights Differences and Similarities of HAE Adult vs. Pediatric Patient Priorities
Love, Work, Progress, and Community in Facioscapulohumeral Muscular Dystrophy
source: pixabay.com

Love, Work, Progress, and Community in Facioscapulohumeral Muscular Dystrophy

Colette Wheeler is one of four siblings, and although each had only a 50/50 chance of inheriting the genetic mutation that leads to facioscapulohumeral muscular dystrophy (FSHD), all of them…

Continue Reading Love, Work, Progress, and Community in Facioscapulohumeral Muscular Dystrophy
Cystic Fibrosis Patients May Now Be Able to Have Hearing Loss Screenings Done Via Tablets
source: pixabay.com

Cystic Fibrosis Patients May Now Be Able to Have Hearing Loss Screenings Done Via Tablets

by Danielle Bradshaw from In The Cloud Copy A recently performed study has demonstrated that it is possible to use tablets to screen patients that have chronic lung conditions -…

Continue Reading Cystic Fibrosis Patients May Now Be Able to Have Hearing Loss Screenings Done Via Tablets
No Link Between Human Papillomavirus Vaccine and Autonomic Dysfunction Syndromes
source: pixabay.com

No Link Between Human Papillomavirus Vaccine and Autonomic Dysfunction Syndromes

by Lauren Taylor from In The Cloud Copy The human papillomavirus (HPV) vaccine has been being administered to teens and young adults worldwide for over a decade now. It has…

Continue Reading No Link Between Human Papillomavirus Vaccine and Autonomic Dysfunction Syndromes
FDA Accepts Investigational New Drug Application for Cervical Dystonia Treatment
source: pixabay.com

FDA Accepts Investigational New Drug Application for Cervical Dystonia Treatment

by Lauren Taylor from In The Cloud Copy Cervical dystonia, sometimes called spasmodic torticollis, is a condition in which the affected individual’s neck muscles involuntarily contract which causes the head…

Continue Reading FDA Accepts Investigational New Drug Application for Cervical Dystonia Treatment