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
Newborn Screening for SMA Appears to Offer Functional Benefits
(MedPage Today) -- Children with spinal muscular atrophy (SMA) identified by newborn screening showed better motor development with disease-modifying treatment than those diagnosed after symptom onset, a German cohort study showed.
Nine out of... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - April 9, 2024 Category: Neurology Source Type: news
Novel biological mechanism discovered that could lead to new treatments for neurological disorders, cancers
The lab of Yongchao C. Ma, PhD, at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago discovered a fundamental biological mechanism that could lead to new treatments for neurological diseases, such as spinal muscular atrophy (SMA) and autism, as well as different cancers. The study was published in the journal Human Molecular Genetics.
Dr. Ma's team found that chemical modification of RNA (called RNA methylation) regulates mitochondrial function. (Source: World Pharma News)
Source: World Pharma News - April 5, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
'I have less than a year left with my baby and I'm exhausted from grieving - but miracle drug will give us more time together as a family'
Ted Chadwick (pictured) had trouble sleeping and feeding after he was born. Then, at six weeks old, his parents learnt these were spinal muscular atrophy type one symptoms. (Source: the Mail online | Health)
Source: the Mail online | Health - March 15, 2024 Category: Consumer Health News Source Type: news
Novartis presents new data on safety and efficacy of Zolgensma
Novartis today presented new data that continue to support the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment. (Source: World Pharma News)
Source: World Pharma News - March 4, 2024 Category: Pharmaceuticals Tags: Featured Novartis Business and Industry Source Type: news
Motor Function in Spinal Muscular Atrophy May Improve With Adjunctive Apitegromab
(MedPage Today) -- Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the phase II TOPAZ trial found... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - February 23, 2024 Category: Neurology 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
Novartis, Voyager sign gene therapy deal worth up to $1.3B
The companies have agreed to a collaboration and license agreement to develop gene therapies for Huntington ’s disease and spinal muscular atrophy. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 2, 2024 Category: Biotechnology Authors: Hannah Green Source Type: news
Why Is Gene Therapy Focused Voyager Therapeutics Stock Soaring Today?
Voyager Therapeutics Inc VYGR announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG NVS, to advance potential gene therapies for Huntington's disease (HD) and spinal muscular atrophy (SMA). Voyager will provide Novartis a…#novartispharmaag #novartisagnvs #sma #voyager #novartis #voyagertracer #alfredsandrock #tracer #aav (Source: Reuters: Health)
Source: Reuters: Health - January 2, 2024 Category: Consumer Health News Source Type: news
This Biotech Just Catapulted 28%, Flashing A Bullish Sign
expanded its gene therapy collaboration with Voyager Therapeutics (VYGR) on Monday. VYGR stock rocketed above its 200-day line for the first time in four months. The two companies will work together to test gene therapies for Huntington's disease and spinal muscular atrophy. Both inherited…#voyagertherapeutics #huntington #voyager #vygr #marketsmithcom #allisongatlin #ibdagatlin #creamofcrop #3steps #avoidbiglosses (Source: Reuters: Health)
Source: Reuters: Health - January 2, 2024 Category: Consumer Health News Source Type: news
These little girls were born with the same cruel genetic illness... but baby Amelia will never suffer like big sister Maisie - thanks to a miracle £75,000-a-dose jab
Portia Thorman of the Spinal Muscular Atrophy charity SMA, said these new treatments were not available when her son Ezra, pictured left, was born. (Source: the Mail online | Health)
Source: the Mail online | Health - December 31, 2023 Category: Consumer Health 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
Sony ’s PlayStation Access controller offers a new social lifeline for gamers with disabilities
Grant Stoner said that he has loved playing video games his entire life, and that his earliest memory is of playing Super Nintendo in his parents’ bedroom at roughly 3 years old. “Gaming, for me, has always been a social activity,” Stoner, a Pittsburgh native who has spinal muscular atrophy type…#grantstoner #supernintendo #stoner #pittsburgh #popsicle #paulamadeuslane #sony #playstation #xbox #adaptivecontroller (Source: Reuters: Health)
Source: Reuters: Health - October 24, 2023 Category: Consumer Health News Source Type: news
