The 'smart suit' that is changing children's lives
Eli Crossley, who has muscular dystrophy, explains now new technology is helping in his daily life. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - February 28, 2023 Category: Consumer Health News Source Type: news
Toxic protein linked to muscular dystrophy and arhinia
NIH researchers discover a possible cause for a rare facial malformation, bringing new hope for patients. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - February 17, 2023 Category: American Health Source Type: news
Toxic Protein Linked to Muscular Dystrophy and Arhinia
Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. (Source: NIEHS News)
Source: NIEHS News - February 17, 2023 Category: Environmental Health Source Type: news
UCLA receives $20 million to establish Goodman –Luskin Microbiome Center
Key takeawaysA $20 million gift from Andrea and Donald Goodman and Renee and Meyer Luskin will fund a new center at UCLA focused on the microbiome and its effect on health.Investigators at the new center will study the microbiome’s role in disease prevention and the body’s immune response.The enterprise will be led by Elaine Hsiao, UCLA ’s De Logi Professor of Biological Sciences.Among the most promising areas of scientific inquiry is the study of the human microbiome and its effect on health. To fuel more rapid progress in this field, Andrea and Donald Goodman and Renee and Meyer Luskin have made a $20 million g...
Source: UCLA Newsroom: Health Sciences - February 15, 2023 Category: Universities & Medical Training Source Type: news
UW spinout raises over $5M to target neuromuscular diseases
Myosana's first target is Duchenne muscular dystrophy, a genetic disorder that leads to muscle degeneration. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 31, 2023 Category: Biotechnology Authors: Rick Morgan Source Type: news
Rockville biotech RegenxBio launches clinical trial in muscular dystrophy
It's one of a series of treatments RegenxBio is developing as it aims to bring multiple products to market by 2025. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - January 24, 2023 Category: American Health Authors: Sara Gilgore Source Type: news
Rockville biotech RegenxBio launches clinical trial in muscular dystrophy
It's one of a series of treatments the company is developing as it aims to bring multiple products to market by 2025. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 23, 2023 Category: Biotechnology Authors: Sara Gilgore Source Type: news
Santhera and ReveraGen Announce FDA Acceptance of New Drug Application for Vamorolone in Duchenne Muscular Dystrophy
Pratteln, Switzerland, and Rockville, MD, USA, January 9, 2023– Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc announce that the U.S. Food and Drug Administration (FDA) has accepted the new drug application (NDA) for... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - January 9, 2023 Category: Drugs & Pharmacology Source Type: news
Drug Trial Snapshot: AMONDYS 45
AMONDYS 45 is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). It is to be used only in patients who have a specific mutation of the dystrophin gene. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - December 2, 2022 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Sarepta Therapeutics Announces That U.S. FDA has Accepted Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta ’s Gene Therapy for Treatment of Ambulant Individuals with Duchenne Muscular Dystrophy
Dear Investor,Please find attached a press release by Sarepta Therapeutics regarding the FDA acceptance of filing and priority review of our partnered DMD asset:https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-us-fda-has-accepted-filing-andDo not hesitate to contact us for any further questions.With best regards,Roche Investor Relations Dr. Bruno EschliPhone: +41 61 68-75284e-mail:bruno.eschli@roche.com Dr. Sabine Borngr äberPhone: +41 61 68-88027e-mail:sabine.borngraeber@roche.com Dr. Birgit MasjostPhone: +41 61 68-84814e-mail:birgit.masjost@roche.comDr. Gerard ...
Source: Roche Investor Update - November 28, 2022 Category: Pharmaceuticals Source Type: news
Sarepta Surges As It Nears The Finish Line With A Muscular Dystrophy Gene Therapy
The Food and Drug Administration said Monday it will perform a speedy review of Sarepta Therapeutics' (SRPT) muscular dystrophy gene therapy, and SRPT stock jumped.X Sarepta filed for approval based on three clinical studies of the gene therapy, SRP-9001. But one study, called Embark, is still…#sareptatherapeutics #sarepta #genetherapy #srpt #gene #sareptagene #therapeutics #therapy #fda #embark (Source: Reuters: Health)
Source: Reuters: Health - November 28, 2022 Category: Consumer Health News Source Type: news
Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta ’s Gene Therapy for the Treatment of Ambulant Individuals with Duchenne Muscular Dystrophy
Regulatory action date of May 29, 2023
SRP-9001 would be the first gene therapy for Duchenne, a one-time treatment designed to treat the underlying cause of DMD by delivering a functional shortened dystrophin to muscle
CAMBRIDGE, Mass., Nov.... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - November 28, 2022 Category: Drugs & Pharmacology Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche provides update on Phase III GRADUATE programme evaluating gantenerumab in early Alzheimer ’s disease
Phase III GRADUATE studies did not meet their primary endpoints of slowing clinical decline in people with early Alzheimer ’sThe level of beta-amyloid removal by gantenerumab was lower than expectedTopline data will be presented at the Clinical Trials on Alzheimer ’s Disease (CTAD) ConferenceRoche is committed to the Alzheimer ’s community and will continue to develop novel diagnostics and potential treatments for Alzheimer’sBasel, 14 November 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the GRADUATE I and II studies evaluating gantenerumab in people with mild cognitive impairment (MCI) du...
Source: Roche Investor Update - November 14, 2022 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche provides update on Phase III GRADUATE programme evaluating gantenerumab in early Alzheimer ’s disease
Phase III GRADUATE studies did not meet their primary endpoints of slowing clinical decline in people with early Alzheimer ’sThe level of beta-amyloid removal bygantenerumab was lower than expectedTopline data will be presented at the Clinical Trials on Alzheimer ’s Disease (CTAD) ConferenceRoche is committed to the Alzheimer ’s community and will continue to develop novel diagnostics and potential treatments for Alzheimer ’sBasel, 14 November 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the GRADUATE I and II studies evaluating gantenerumab in people with mild cognitive impairment (MCI) du...
Source: Roche Investor Update - November 14, 2022 Category: Pharmaceuticals Source Type: news
