UCLA Samueli to lead $4 million cell research project funded by Chan Zuckerberg Initiative
Key takeawaysThe grant will fund a three-year collaboration among researchers at UCLA, USC and Caltech to advance cell and tissue engineering technologies.The project, led by UCLA ’s Dino Di Carlo, will engineer, manipulate and analyze cell-to-cell interactions that underlie complex multicellular systems in the body.Di Carlo said he aims for the collaboration to develop into a long-term partnership across institutions to advance biotechnology in Los Angeles.The Chan Zuckerberg Initiativetoday announced a $4 million grant to support research led by the UCLA Samueli School of Engineering that will examine cellular behavio...
Source: UCLA Newsroom: Health Sciences - February 29, 2024 Category: Universities & Medical Training Source Type: news
Drug Trials Snapshots: AGAMREE
AGAMREE is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - February 16, 2024 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Invitation to Roche ’s Virtual Neurology Investor Event
We are pleased to invite investors and analysts to participate in our virtual event onMonday,11 March 2024, highlighting key data presented at 2024 Muscular Dystrophy Association (MDA) and Alzheimer ' s& Parkinson ' s Diseases (AD/PD) conferences. 15:00 – 16:30 CET / 14:00 – 15:30 GMT10:00 – 11:30 AM EDT / 07:00 – 08:30 AM PDT The webinar will start with presentations, followed by a Q&A session (live access to the speakers). The slides will be available for download at 14:00 CET on the day of the event.>click here Access to virtual event (pre-registration required)Please pre-register for our webin...
Source: Roche Investor Update - February 12, 2024 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche exceeds guidance and achieves sales growth of 1% (CER) for 2023 despite sharp COVID-19 sales decline
Group salesgrow by 1%1 at constant exchange rates (CER; -7% in CHF), more than offsetting the decline in COVID-19-related sales and biosimilar erosion, and thereby exceeding 2023 guidanceExcluding COVID-19 products,Group sales increase by 8%Pharmaceuticals Divisionsalesincrease by 6% (excluding COVID-19 medicine Ronapreve: +9%) due to ongoing high demand for newer medicines, with eye medicine Vabysmo continuing to be the top growth driver, followed by Ocrevus (multiple sclerosis), Hemlibra (haemophilia A) and Polivy (blood cancer)Diagnostics Division salesare 13% lower due to high demand for COVID-19 tests in 2022; strong ...
Source: Roche Media News - February 1, 2024 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche exceeds guidance and achieves sales growth of 1% (CER) for 2023 despite sharp COVID-19 sales decline
Group salesgrow by 1%1 at constant exchange rates (CER; -7% in CHF), more than offsetting the decline in COVID-19-related sales and biosimilar erosion, and thereby exceeding 2023 guidanceExcluding COVID-19 products,Group sales increase by 8%Pharmaceuticals Divisionsalesincrease by 6% (excluding COVID-19 medicine Ronapreve: +9%) due to ongoing high demand for newer medicines, with eye medicine Vabysmo continuing to be the top growth driver, followed by Ocrevus (multiple sclerosis), Hemlibra (haemophilia A) and Polivy (blood cancer)Diagnostics Division salesare 13% lower due to high demand for COVID-19 tests in 2022; strong ...
Source: Roche Investor Update - February 1, 2024 Category: Pharmaceuticals Source Type: news
PTC Therapeutics Recovers From An Ugly Fall After European Officials Reject DMD Drug
PTC stock rose Friday after European regulators voted to withdraw the biotech company's treatment for a severe form of muscular dystrophy. The post PTC Therapeutics Recovers From An Ugly Fall After European Officials Reject DMD Drug appeared first on Investor's Business Daily. (Source: Reuters: Health)
Source: Reuters: Health - January 27, 2024 Category: Consumer Health News Source Type: news
In Early Research, Heart Drugs Show Promise in Fighting Muscular Dystrophy
WEDNESDAY, Jan. 3, 2024 -- A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests.Myotonic dystrophy type 1 (DM1) is caused by abnormal RNA... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - January 3, 2024 Category: General Medicine Source Type: news
Why Is Neuromuscular Disease Focused Entrada Therapeutics Shares Trading Lower Today?
Entrada Therapeutics Inc TRDA shares are trading lower after the company announced updates on ENTR-601-44 in Duchenne Muscular Dystrophy. The FDA previously notified Entrada that the Investigational New Drug (IND) application for the Phase 1 clinical trial of ENTR-601-44 had been placed on…#fda #entrada #dipaldoshi #entradatherapeutics #williamblair (Source: Reuters: Health)
Source: Reuters: Health - November 22, 2023 Category: Consumer Health News Source Type: news
Children ’s National Hospital spinout ReveraGen BioPharma gearing up for muscular dystrophy drug launch
ReveraGen BioPharma just won FDA approval for a treatment it's been developing for 15 years. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - November 14, 2023 Category: Pharmaceuticals Authors: Sara Gilgore Source Type: news
' Hidden' Cognitive Impairments in DMD May Worsen Outcomes'Hidden' Cognitive Impairments in DMD May Worsen Outcomes
A natural history study that utilized the Pediatric Quality of Life assessment showed that patients with Duchenne muscular dystrophy reported the lowest scores in brain health, including emotional health and school performance.MDedge News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 8, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Government plans to offer support for treatment of rare diseases
"Accordingly, as these patients have already commenced treatment, Sarepta Therapeutics has committed, as per the communication received by the NDRC (National Rare Diseases' Committee), to continue treating these patients with Duchenne muscular dystrophy for the next three months, and to supply the medication free of cost during this period," it said. The court directed doctor Madhulika Kabra from the All India Institute of Medical Science to place the order for the medicine with the company within one week and expedite the procurement for these 14 patients. (Source: The Economic Times)
Source: The Economic Times - November 7, 2023 Category: Consumer Health News Source Type: news
DMD Gene Therapy Safe, Effective at 4 Years DMD Gene Therapy Safe, Effective at 4 Years
Four years after a single dose of SRP-9001 gene therapy, four children with Duchenne muscular dystrophy show sustained stabilization of motor function that would otherwise show expected decline.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 6, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints
This study is ongoing.Study 301 (EMBARK), a Phase 3 global, randomised, double-blinded and placebo-controlled study of Elevidys in ambulatory Duchenne patients aged 4-<8 years old.The ENVOL trial (Study 302) a Phase 2 study in children with Duchenne. The study aims to enrol 21 participants who are under 4 years of age, including newborns. Not yet started.The ENVISION trial (Study 303), a Phase 3 study in older ambulatory/non-ambulatory patients which is now recruiting.The EXPEDITION long-term (5 year) follow up study (Study 305) of participants who have received Elevidys in a previous clinical study, which is not yet re...
Source: Roche Media News - October 31, 2023 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints
This study is ongoing.Study 301 (EMBARK), a Phase 3 global, randomised, double-blinded and placebo-controlled study of Elevidys in ambulatory Duchenne patients aged 4-<8 years old.The ENVOL trial (Study 302) a Phase 2 study in children with Duchenne. The study aims to enrol 21 participants who are under 4 years of age, including newborns. Not yet started.The ENVISION trial (Study 303), a Phase 3 study in older ambulatory/non-ambulatory patients which is now recruiting.The EXPEDITION long-term (5 year) follow up study (Study 305) of participants who have received Elevidys in a previous clinical study, which is not yet re...
Source: Roche Investor Update - October 31, 2023 Category: Pharmaceuticals Source Type: news
Sarepta ’s Duchenne gene therapy fails to meet main goal in key trial
The Cambridge biotech said late Monday that its gene therapy to treat Duchenne muscular dystrophy failed to meet the primary goal of a large Phase 3 trial, but that it plans to seek full approval regardless. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 31, 2023 Category: Pharmaceuticals Authors: Don Seiffert Source Type: news
