Updates in Parkinson's, ICH, Migraine, Muscular Dystrophy Updates in Parkinson's, ICH, Migraine, Muscular Dystrophy
Prof Christoph Diener discusses his picks for updates in neurology that were published in April 2024.Medscape Neurology (Source: Medscape Pharmacist Headlines)
Source: Medscape Pharmacist Headlines - May 23, 2024 Category: Drugs & Pharmacology Tags: Neurology & Neurosurgery Commentary Source Type: news

Second Death - Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial
A young patient died due to cardiac arrest after receiving Pfizer Inc’s PFE experimental gene therapy in a mid-stage trial for Duchenne muscular dystrophy (DMD), the company told in a letter to Parent Project Muscular Dystrophy. The boy was enrolled in Phase 2 DAYLIGHT study studying the treatment…#pfizerincs #pfe #dmd #musculardystrophy #citingpfizer #clinicaltrialgov #pfizer #ciffreo #fda (Source: Reuters: Health)
Source: Reuters: Health - May 8, 2024 Category: Consumer Health News Source Type: news

Young participant in Pfizer's DMD gene therapy trial dies
A young boy with Duchenne muscular dystrophy (DMD) participating in Pfizer’s phase 2 gene therapy trial has died, the pharma shared in a May 7 letter (PDF) cited by nonprofit Parent Project Muscular Dystrophy. The boy had received Pfizer’s fordadistrogene movaparvovec, an investigational…#dmd #pfizer #pdf #daylight #ciffreo #mikaeldolsten #phd #sareptatherapeutics #pf06939926 #fda (Source: Reuters: Health)
Source: Reuters: Health - May 7, 2024 Category: Consumer Health News Source Type: news

Drug watchdog criticizes Sarepta gene therapy approval, cost
The chief medical officer of ICER wrote that the price for the company's gene therapy intended to treat Duchenne muscular dystrophy is “enormous” considering it "failed to meet its primary end point." (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 3, 2024 Category: Biotechnology Authors: Hannah Green Source Type: news

Investigational FSHD Drug Did Little to Change DUX4 Expression
(MedPage Today) -- The investigational drug losmapimod did not significantly change expression of the gene behind facioscapulohumeral muscular dystrophy (FSHD), but it was associated with potential improvements in select structural and patient... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - May 2, 2024 Category: Neurology Source Type: news

Roche ’s OCREVUS subcutaneous injection receives EU CHMP positive opinion for relapsing and primary progressive multiple sclerosis
If approved, OCREVUS subcutaneous (SC) would be the first and only twice-a-year, 10-minute injection for relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS) in the EUOCREVUS SC will be a new, faster way of administering OCREVUS with a comparable efficacy and safety profile tothe well-established intravenous (IV) infusionOCREVUS SC has the potential to expand treatment optionsto centres without IV infrastructure or with IV constraintsBasel, 29 April 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency ’s Committee for Medicinal Products for Human Use...
Source: Roche Investor Update - April 29, 2024 Category: Pharmaceuticals Source Type: news

DMD: What Are the Measures That Matter? DMD: What Are the Measures That Matter?
Watch this exciting on-demand symposium, presented by Dr Tina Duong and Prof Laurent Servais, to learn more about functional assessment tools used in Duchenne muscular dystrophy, as well as when and how we should use these tools.Medscape Medical Affairs (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - April 26, 2024 Category: Consumer Health News Tags: None Virtual Symposium Source Type: news

Roche ’s subcutaneous OCREVUS one-year data demonstrates near-complete suppression of clinical relapses and brain lesions in patients with progressive and relapsing forms of MS
Results from the Phase III study showed that subcutaneous (SC) injection was consistent with IV infusion and demonstrated near-complete suppression of relapse activity (97%) and MRI lesions (97.2%) through 48 weeksThe twice-yearly, 10-minute SC injection has the potential to expand the usage of OCREVUS to treatment centres without IV infrastructure or with IV capacity limitationsU.S. FDA and EMA accepted filings based on the data from OCARINA II, with EU approval anticipated mid-2024 and U.S. approval anticipated September 2024Basel, 17 April 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today data from the Phase III...
Source: Roche Media News - April 17, 2024 Category: Pharmaceuticals Source Type: news

Possible Pulmonary Benefit With Viltolarsen for Duchenne Muscular Dystrophy
(MedPage Today) -- DENVER -- Viltolarsen (Viltepso) was well tolerated by boys and men with Duchenne muscular dystrophy (DMD), with no new safety signals, according to results from the open-label, phase II Galactic53 trial. The results also... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - April 16, 2024 Category: Neurology Source Type: news

Jack was given pioneering drink to beat muscle wasting disease and can still stand. His best friend Eli didn't get the drug and now he is permanently in a wheelchair. Their contrasting stories prove new treatment works, so why is NHS rollout at risk?
Eli Crossley and Jack Johnson (pictured) both have Duchenne muscular dystrophy (DMD) -progressive muscle-wasting disease. (Source: the Mail online | Health)
Source: the Mail online | Health - April 6, 2024 Category: Consumer Health News Source Type: news

Givinostat Slows Duchenne Muscular Dystrophy Decline
(MedPage Today) -- Givinostat (Duvyzat) slowed physical decline as measured by stair climbing speed for a subset of boys with Duchenne muscular dystrophy (DMD), according to the phase III trial that supported the drug's recent approval for this... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - April 5, 2024 Category: Neurology Source Type: news

COVID-Cautious Americans Feel Abandoned
For all of 2020, Alex, a 28-year-old living in New York, followed the U.S. Centers for Disease Control and Prevention’s (CDC) COVID-19 guidance “religiously.” Then, in 2021, something began to shift. That spring, the CDC said it was okay for vaccinated people to ditch their masks in most places. But people were clearly still getting sick—including Alex, who got COVID-19 for the first time in late 2021 and later developed Long COVID symptoms. [time-brightcove not-tgx=”true”] “There was this reckoning moment where it was like, ‘Maybe the CDC is not being totally honest with...
Source: TIME: Health - March 27, 2024 Category: Consumer Health News Authors: Jamie Ducharme Tags: Uncategorized COVID-19 healthscienceclimate Source Type: news

Living With Muscular Dystrophy at 50 Makes Death My Shadow Partner
I am an Aries through and through—bold, ambitious, fiery, and confident.  Today, March 27, marks my 50th year on this planet, something I could never have imagined. I was diagnosed with an undetermined type of muscular dystrophy as a young child, and doctors told my parents I wouldn’t live to become an adult. My immigrant parents cried when they heard the news. Even though this news was devastating, they never treated me like a fragile egg about to break. In fact, as the first born child of three girls, I had a lot of responsibilities and expectations which only reinforced my Aries tendencies. [time-bright...
Source: TIME: Health - March 27, 2024 Category: Consumer Health News Authors: Alice Wong Tags: Uncategorized freelance Source Type: news

FDA Approves Oral Duvyzat for Duchenne Muscular Dystrophy
MONDAY, March 25, 2024 -- The U.S. Food and Drug Administration has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.Duvyzat is a histone deacetylase... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - March 25, 2024 Category: Pharmaceuticals Source Type: news

FDA approves nonsteroidal treatment for Duchenne muscular dystrophy
The U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle. (Source: World Pharma News)
Source: World Pharma News - March 25, 2024 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news