Cannabidiol in Children and Young Adults With Rare Disease-associated Severe Epilepsy
Conditions: Epilepsy; Rare Diseases Intervention: Drug: Cannabidiol oral solution Sponsor: Meyer Children's Hospital IRCCS Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 7, 2023 Category: Research Source Type: clinical trials
Therapy for Rare Bone Disorder Shows Promise in NIH Clinical Trial
April 4, 2023 -- A clinical trial at the National Institutes of Health found that a medication, denosumab, significantly reduced abnormal bone turnover in adults with fibrous dysplasia, a rare disease marked by weak and misshapen bones. Bone... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - April 4, 2023 Category: Pharmaceuticals Source Type: clinical trials
BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis
DiscussionThe BEAT CF PEx cohort will report clinical, treatment, and outcome data for PEx among people with CF and is intended to serve as a core (master) protocol for future nested, interventional trials evaluating treatment(s) for these episodes. The protocols for nested sub-studies are beyond the scope of this document and will be reported separately.Trial registrationANZCTR BEAT CF Platform – ACTRN12621000638831. Registration date: Sept. 26, 2022. (Source: Trials)
Source: Trials - March 22, 2023 Category: Research Source Type: clinical trials
Announces Research Breakthrough with JOTROL in Parkinson's Disease
JUPITER, Fla. and BOSTON, Feb. 27, 2023. Jupiter Neurosciences, Inc. ( " Jupiter " or the " Company " ), a clinical-stage pharmaceutical company advancing a pipeline focused on the treatment of central nervous system (CNS) disorders and rare diseases,... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - February 27, 2023 Category: Pharmaceuticals Source Type: clinical trials
Functional Tests to Resolve Unsolved Rare Diseases. Rares.
Conditions: Intellectual Disability; Rubinstein-Taybi Syndrome; Cystic Fibrosis; Congenital Heart Defect; Periventricular Nodular Heterotopia; Neurodegeneration With Brain Iron Accumulation (NBIA); Albinism Interventions: Genetic: Ex-vivo approach concerning 25 patients; Genetic: In-vitro approach concerning 25 patients Sponsor: University Hospital, Bordeaux Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 25, 2023 Category: Research Source Type: clinical trials
