Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Evrysdi ® (risdiplam) data further confirm long-term efficacy and safety data in a broad range of people with spinal muscular atrophy (SMA)Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to sit, stand and walk within timeframes typical of healthy babiesNew gene therapy data in boys living with Duchenne muscular dystrophy (DMD) reports encouraging results from early stage study supporting its progress into global pivotal Phase III EMBARK studyBasel, 08 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data...
Source: Roche Media News - March 8, 2022 Category: Pharmaceuticals Source Type: news
Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio
Evrysdi ® (risdiplam) data further confirm long-term efficacy and safety data in a broad range of people with spinal muscular atrophy (SMA)Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to sit, stand and walk within timeframes typical of healthy babiesNew gene therapy data in boys living with Duchenne muscular dystrophy (DMD) reports encouraging results from early stage study supporting its progress into global pivotal Phase III EMBARK studyBasel, 08 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data...
Source: Roche Investor Update - March 8, 2022 Category: Pharmaceuticals Source Type: news
Roche and three leading research institutions combine expertise on new Alzheimer ’s prevention trial
Phase III trial will evaluategantenerumab in cognitively unimpaired people with the earliest biologicalsigns ofAlzheimer ' sdiseaseRoche and research institutions have jointly leveraged their scientific expertise on Alzheimer ’s disease to design the new studyBasel, 3 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced a new Phase III Alzheimer ’s disease prevention trial (SKYLINE, NCT05256134) with gantenerumab, an investigational anti-amyloid antibody that is administered subcutaneously. Roche intends to enter into a collaboration agreement with Banner Alzheimer’s Institute’s Alzheimer’s Prevention ...
Source: Roche Investor Update - March 3, 2022 Category: Pharmaceuticals Source Type: news
AMRA gets U.S., Canadian clearance for MRI-based body composition scan
Swedish image analysis software developer AMRA Medical has secured clearanc...Read more on AuntMinnie.comRelated Reading:
AMRA Medical gets FDA clearance for MasS Scan
Canada greenlights distribution of AMRA's muscle scan
AMRA touts liver MRI research at AASLD show
AMRA joins clinical trial on muscular dystrophy
AMRA touts clinical trial results (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - February 23, 2022 Category: Radiology Source Type: news
Invitation to Roche ’s virtual event on key neuromuscular data presented at the 2022 Muscular Dystrophy Association (MDA) scientific and clinical conference
We are pleased to invite investors and analysts to participate in our virtual event onWednesday, 16 March 2022, highlighting Roche data presented during the 2022 Muscular Dystrophy Association - MDA -Virtual Clinical and Scientific Conference, from 13th to 16th March.16:30 – 17:30 CET / 15:30 - 16:30 GMT11:30 – 12:30 am EDT / 8:30 – 9:30 am PDT The webinar will start with a presentation, followed by a Q&A session (live access to the speakers). The slides will be available fordownload at15:30 CET on the day of the event.>click here Access to virtual event (pre-registration required)Please pre-register for o...
Source: Roche Investor Update - February 22, 2022 Category: Pharmaceuticals Source Type: news
Muscular dystrophy affects 40,000 more people in the UK - study
THE number of people in the UK with muscle wasting conditions may be 40,000 higher than previously thought, a charity has said. (Source: Daily Express - Health)
Source: Daily Express - Health - February 15, 2022 Category: Consumer Health News Source Type: news
Roche ’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spinal muscular atrophy (SMA)
Basel, 25 January 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). The sNDA submission incorporates interim data from the RAINBOWFISH study, which shows the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting, standing, walking and maintained the ability to swallow following 12 months of treatment. “Treating very youn...
Source: Roche Media News - January 25, 2022 Category: Pharmaceuticals Source Type: news
Roche ’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spinal muscular atrophy (SMA)
Basel, 25 January 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). The sNDA submission incorporates interim data from the RAINBOWFISH study, which shows the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting, standing, walking and maintained the ability to swallow following 12 months of treatment. “Treating very youn...
Source: Roche Investor Update - January 25, 2022 Category: Pharmaceuticals Source Type: news
Roche ’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA)
Interim data submitted to the FDA show majority of pre-symptomatic babies treated withEvrysdi for at least one year were able to sit, stand and walk within timeframes typical of healthy babies, as well as maintain swallowingEvrysdi is approved in 70 countries and submitted in a further 31 with more than 4,500 patients treated to dateBasel, 25 January 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age w...
Source: Roche Media News - January 25, 2022 Category: Pharmaceuticals Source Type: news
Roche ’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spinal muscular atrophy (SMA)
Interim data submitted to the FDA show majority of pre-symptomatic babies treated withEvrysdi for at least one year were able to sit, stand and walk within timeframes typical of healthy babies, as well as maintain swallowingEvrysdi is approved in 70 countries and submitted in a further 31 with more than 4,500 patients treated to dateBasel, 25 January 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age w...
Source: Roche Investor Update - January 25, 2022 Category: Pharmaceuticals Source Type: news
What Are Some Initial Evaluations for Suspected Congenital Muscle Diseases?
Discussion
Usually congenital myopathies (CM, e.g. nemaline, core, centronuclear myopathies, etc.) and congenital muscular dystrophy (CMD, e.g. LAMA2-related, collage VI-related, alpha-dystroglycan-related muscular dystrophy, etc.) have been diagnosed based on physical examination and histopathology. Traditionally CMs are due to problems with the muscle contractile apparatus and structures that assist excitation-contraction coupling. CMD are due to problems with the extracellular matrix, muscle membrane and sarcolemmal membrane. Differentiating between CM and CMD have become more blurred as genetic testing and additional r...
Source: PediatricEducation.org - January 17, 2022 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
'Unmovable monster': The drug trial that changed how biotechs approach rare disease treatments
Billy Ellsworth was 10 years old when he started traveling from his home outside Philadelphia to Columbus, Ohio, to take part in a small trial of an experimental drug to treat the rare, degenerative disease with which he was born.
A decade later, his memories of that time are fleeting. He remembers knowing the drug was intended to help his muscles to stay healthy despite the effects of Duchenne muscular dystrophy. He remembers being nervous about his first biopsy, having never undergone surgery… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - December 26, 2021 Category: Health Management Authors: Don Seiffert Source Type: news
'Unmovable monster': The drug trial that changed how biotechs approach rare disease treatments
Billy Ellsworth was 10 years old when he started flying from his home outside Philadelphia to Columbus, Ohio to take part in a small trial of an experimental drug to treat the rare, degenerative disease with which he was born.
A decade later, his memories of that time are fleeting. He remembers knowing the drug was intended to help his muscles to stay healthy despite the effects of Duchenne muscular dystrophy. He remembers being nervous about his first biopsy, having never undergone surgery before.… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - December 23, 2021 Category: American Health Authors: Don Seiffert Source Type: news
Q & A: Dr. Thomas Rando on preventing age-related diseases and turning discoveries into cures
For Dr. Thomas Rando, the path to becoming a physician-scientist began with something that hedidn ’t learn in high school biology.After one class that touched on the connections between neurons and muscle fibers, Rando took it upon to himself to find all the information he could about how cells communicate through electrical signals.Soon, he began pursuing that interest at Harvard University, where he completed his undergraduate work, a doctorate in cell and developmental biology and his medical degree.Rando joined the neurology faculty at the Stanford University School of Medicine in 1995.There, he founded a clinic to t...
Source: UCLA Newsroom: Health Sciences - December 10, 2021 Category: Universities & Medical Training Source Type: news
AMRA Medical gets FDA clearance for MasS Scan
Swedish image analysis software developer AMRA Medical has received U.S. Foo...Read more on AuntMinnie.comRelated Reading:
Canada greenlights distribution of AMRA's muscle scan
AMRA touts liver MRI research at AASLD show
AMRA joins clinical trial on muscular dystrophy
AMRA touts clinical trial results
AMRA adds UK Biobank images to database (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - December 7, 2021 Category: Radiology Source Type: news
