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
In ‘Killers of the Flower Moon,’ Is Leonardo DiCaprio Playing a Dumb Hick, a Pitiless Sociopath…or a Muddle?
A movie’s central character needn’t be someone we admire, but he should probably be someone we’re drawn to, someone we vibe with in sympathetic fascination, who we feel we know and understand even as he crosses over to the dark side. Few movies have lived out that dynamic more cathartically than…#martinscorsese #meanstreets #scorsese #harveykeitels #robertdeniros #johnnyboy #deniros #travisbickle #goodfellas #rayliotta (Source: Reuters: Health)
Source: Reuters: Health - October 29, 2023 Category: Consumer Health News Source Type: news
Will the NHS fund a £2.6m life-changing gene drug jab that allows boys with a rare muscle wasting disease the chance of walking
John Paul Hopkins, 32, from North Wales is hoping to get his son Rory, 6, who was diagnosed with Duchenne muscular dystrophy in 2021, onto gene therapy like Elevidys. (Source: the Mail online | Health)
Source: the Mail online | Health - October 28, 2023 Category: Consumer Health News Source Type: news
FDA Okays Drug for Duchenne Muscular Dystrophy FDA Okays Drug for Duchenne Muscular Dystrophy
Vamorolone (Agamree) is a structurally unique steroidal anti-inflammatory drug with fewer side effects than traditional corticosteroids.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - October 27, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news
Novel Corticosteroid Approved for Rare Disease
(MedPage Today) -- Vamorolone (Agamree), a novel corticosteroid treatment, won FDA approval to treat Duchenne muscular dystrophy (DMD) in patients ages 2 years and older, Catalyst Pharmaceuticals announced Thursday.
The dissociative steroidal... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - October 27, 2023 Category: American Health Source Type: news
FDA Approves Agamree (vamorolone) for the Treatment of Duchenne Muscular Dystrophy
CORAL GABLES, Fla., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ( " Catalyst " or " Company " ) (Nasdaq: CPRX) today reported that Santhera Pharmaceuticals ( " Santhera " ) has obtained U.S. Food and Drug Administration ( " FDA " ) approval... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - October 26, 2023 Category: Drugs & Pharmacology Source Type: news
Opening Our Eyes To Stem Cells For Stargardt's Macular Dystrophy
Stem cell therapies are being studied in clinical trials to treat retinitis pigmentosa, a genetic disorder that causes vision loss. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - October 26, 2023 Category: Pharmaceuticals Authors: William A. Haseltine, Contributor Tags: Healthcare /healthcare Innovation /innovation business pharma & standard Source Type: news
Digital Sanctions: Israel Disables Over 100 Binance Accounts Potentially Linked to Hamas
Hamas has been leveraging a global financial network to channel support from charities and sympathetic nations, employing various methods, including cryptocurrencies, to circumvent international sanctions. This has prompted the Israeli authorities to freeze several crypto accounts and seize…#hamas #israeli #israelfreezesover100 #binance #cryptocurrencies #tomalexandrovich #israel #cftc #ak47 #tetherfreezes (Source: Reuters: Health)
Source: Reuters: Health - October 17, 2023 Category: Consumer Health News Source Type: news
What Causes Dysphagia?
Discussion
Swallowing is a complex process with 4 phases:
Preparatory – food is moistened with saliva, chewed and prepared into a bolus by teeth, tongue and hard palate.
Oral – food bolus is moved into oropharynx by tongue and triggers the swallow reflex. Soft palate elevates to prevent nasopharyngeal reflux.
Pharyngeal – food bolus is moved through the oropharynx and hypopharynx to the esophagus. Respiration stops briefly with vocal fold adduction and larynx elevation to prevent aspiration.
Esophageal – the cricopharyngeaul muscle relaxes which allows the food bolus into the esophagus where it is ...
Source: PediatricEducation.org - October 16, 2023 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
How hackers piled onto the Israeli-Hamas conflict
Hackers sympathetic to Hamas are working to make the Israel-Gaza conflict the next front of cyberwarfare. Hacking groups with links to countries including Iran and Russia have launched a series of cyberattacks and online campaigns against Israel over the past week, some that may have even occurred…#hamas #israel #iran #telegram #israeli #jerusalempost #ukraine #lizwu #checkpointsoftware #gaza (Source: Reuters: Health)
Source: Reuters: Health - October 15, 2023 Category: Consumer Health News Source Type: news
A disabled Israeli teenager with muscular dystrophy is a hostage held by Hamas in Gaza after she was abducted from the Supernova music festival
Disabled hostage Rut Perez and her father, Eric Perez, who has also been missing since Hamas' attacks. Amit Azriel Hamas militants took a disabled 17-year-old hostage who attended the Supernova music festival. Rut Perez is a highly vulnerable wheelchair user with muscular dystrophy. Hamas has…#rutperez #ericperez #hamas #amitazriel #supernova #israelis #gaza #timesoflondon #times #israeli (Source: Reuters: Health)
Source: Reuters: Health - October 14, 2023 Category: Consumer Health News Source Type: news
Late-breaking data for Roche ’s BTK inhibitor fenebrutinib show brain penetration and significant reduction in lesions in patients with relapsing multiple sclerosis
New data from Phase II FENopta study in relapsing multiple sclerosis (RMS) show fenebrutinib crosses the blood-brain barrier with the potential to act directly on the chronic inflammation related to multiple sclerosis (MS)More than 90% relative reduction in new/enlarging T2 lesions and new T1 gadolinium-enhancing (Gd+) lesions with fenebrutinib beginning at 8 weeksThe safety profile of fenebrutinib was consistent with previous and ongoing clinical trials across more than 2,500 people to dateBasel, 13 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data from the Phase II FENopta study showing that inve...
Source: Roche Media News - October 13, 2023 Category: Pharmaceuticals Source Type: news
Late-breaking data for Roche ’s BTK inhibitor fenebrutinib show brain penetration and significant reduction in lesions in patients with relapsing multiple sclerosis
New data from Phase II FENopta study in relapsing multiple sclerosis (RMS) show fenebrutinib crosses the blood-brain barrier with the potential to act directly on the chronic inflammation related to multiple sclerosis (MS)More than 90% relative reduction in new/enlarging T2 lesions and new T1 gadolinium-enhancing (Gd+) lesions with fenebrutinib beginning at 8 weeksThe safety profile of fenebrutinib was consistent with previous and ongoing clinical trials across more than 2,500 people to dateBasel, 13 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data from the Phase II FENopta study showing that inve...
Source: Roche Investor Update - October 13, 2023 Category: Pharmaceuticals Source Type: news
New data for Roche ’s OCREVUS show that after 10 years of treatment 77% of people with relapsing multiple sclerosis were free from disability progression and 92% continue to walk unaided
10-year efficacy data highlight OCREVUS ’ impact on preventing disability progression and maintaining mobility in both relapsing and progressive forms of multiple sclerosis (MS)10-year safety data from over 6,000 patients continue to reinforce consistent long-term safety profile of OCREVUSMore than 3,200 women with MS treated with OCREVUS reported no increased risk in adverse pregnancy and infant outcomes with real-world analyses showing low risk of relapse during and after pregnancyOCREVUS controlled disease activity and progression over one year in Black and Hispanic / Latinx people with MSBasel, 12 October 2023 - Roch...
Source: Roche Media News - October 12, 2023 Category: Pharmaceuticals Source Type: news
