Neurogenetic disorders

SadBaby is Opening New Doorways for Providing Financial Aid to SMA Patients

Retrieved on: 
Thursday, July 29, 2021
Neurological disorders, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Spinal muscular atrophy, SMA

ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.

Key Points: 
  • ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.
  • The SadBaby Team aims to improve every patient's quality of life and gather sufficient funds to support the affected children and their families.
  • Working together with SMA Association in Antalya, SadBaby has already raised significant donations for babies affected with SMA and is soon organising a concert to further their funding strategy.
  • SadBaby is also planning to create an NFT marketplace where families would be able to sell NFT products and obtain financial help by this year's end.

SadBaby is Opening New Doorways for Providing Financial Aid to SMA Patients

Retrieved on: 
Thursday, July 29, 2021
Neurological disorders, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Spinal muscular atrophy, SMA

ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.

Key Points: 
  • ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.
  • The SadBaby Team aims to improve every patient's quality of life and gather sufficient funds to support the affected children and their families.
  • Working together with SMA Association in Antalya, SadBaby has already raised significant donations for babies affected with SMA and is soon organising a concert to further their funding strategy.
  • SadBaby is also planning to create an NFT marketplace where families would be able to sell NFT products and obtain financial help by this year's end.

Data for Genentech’s Evrysdi (risdiplam) Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies With Type 1 Spinal Muscular Atrophy (SMA)

Retrieved on: 
Wednesday, July 28, 2021
Research, Baby, Maternity, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Consumer, Health, Science, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, SMA, Muscle atrophy, Spinal muscular atrophies, Evrysdi® (risdiplam), SMA, Genentech in Neuroscience, Genentech, EVRYSDI® (RISDIPLAM), SMA, GENENTECH IN NEUROSCIENCE, GENENTECH

Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.

Key Points: 
  • Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.
  • We will continue to work closely with governments and the SMA community to bring Evrysdi to as many people as possible.
  • Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
  • If Evrysdi gets in ones eyes, rinse ones eyes with water
    The most common side effects of Evrysdi include:

SadBaby launches Crowdfund Platform for Aiding SMA Patients

Retrieved on: 
Sunday, July 11, 2021
Neurogenetic disorders

The project launched by SadBaby focuses on gathering funds through their supporters and investors and utilizes it for meeting the financial requirements of the SMA patients.

Key Points: 
  • The project launched by SadBaby focuses on gathering funds through their supporters and investors and utilizes it for meeting the financial requirements of the SMA patients.
  • Sadbaby Token is developed to encourage holders for making donations and contributing at any level as per their desire.
  • The main goal of SadBaby is to help all the babies affected with SMA.
  • SadBaby aims to donate financially to SMA patients and help them fight the disease with support and donations from its community and angel investors.

SadBaby launches Crowdfund Platform for Aiding SMA Patients

Retrieved on: 
Sunday, July 11, 2021
Neurogenetic disorders

The project launched by SadBaby focuses on gathering funds through their supporters and investors and utilizes it for meeting the financial requirements of the SMA patients.

Key Points: 
  • The project launched by SadBaby focuses on gathering funds through their supporters and investors and utilizes it for meeting the financial requirements of the SMA patients.
  • Sadbaby Token is developed to encourage holders for making donations and contributing at any level as per their desire.
  • The main goal of SadBaby is to help all the babies affected with SMA.
  • SadBaby aims to donate financially to SMA patients and help them fight the disease with support and donations from its community and angel investors.

Scholar Rock to Present Apitegromab TOPAZ Phase 2 Trial Results Highlighting Pharmacokinetic (PK) and Pharmacodynamic (PD) Data at the 2021 European Academy of Neurology Congress

Retrieved on: 
Friday, June 18, 2021
Health, Other Health, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Muscular dystrophy, Myostatin, SMA, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

Scholar Rock plans to initiate a Phase 3 trial to further evaluate apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA by the end of 2021.

Key Points: 
  • Scholar Rock plans to initiate a Phase 3 trial to further evaluate apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA by the end of 2021.
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
  • Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path.

Muscular Dystrophy Association Launches Quest Podcast Hosted By Motivational Speaker and Author Mindy Henderson

Retrieved on: 
Monday, June 14, 2021
Neurological disorders, Branches of biology, Neurodegenerative disorders, Neurogenetic disorders, Neuromuscular Disorders, Motor neuron diseases, Amyotrophic lateral sclerosis, Muscular Dystrophy Association, Neuromuscular disease, Rare diseases, Dystrophy, Muscular dystrophy

The podcast will be hosted by Mindy Henderson , who lives with neuromuscular disease and is a motivational speaker, author, and hostof " The Truth About Things That Suck " podcast.

Key Points: 
  • The podcast will be hosted by Mindy Henderson , who lives with neuromuscular disease and is a motivational speaker, author, and hostof " The Truth About Things That Suck " podcast.
  • Henderson was diagnosed with spinal muscular atrophy (SMA) when she was 15 months old.
  • "I'm thrilled to be hosting the new Quest Podcast," said Henderson.
  • For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases.

Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 and 3 Spinal Muscular Atrophy at the 2021 Virtual SMA Research & Clinical Care Meeting

Retrieved on: 
Monday, June 7, 2021
FDA, Health, Genetics, General Health, Pharmaceutical, Biotechnology, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Myostatin, SMA, Spinal muscular atrophies, SMA4, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.

Key Points: 
  • A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
  • Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children.

Scholar Rock Receives Fast Track Designation from the U.S. FDA for Apitegromab for the Treatment of Patients with Spinal Muscular Atrophy

Retrieved on: 
Monday, May 24, 2021
FDA, Health, Genetics, General Health, Pharmaceutical, Biotechnology, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Muscular dystrophy, Myostatin, Spinal muscular atrophy, SMA, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

Through Fast Track, Scholar Rock is eligible to submit a rolling Biologic License Application (BLA) for apitegromab if relevant criteria are met.

Key Points: 
  • Through Fast Track, Scholar Rock is eligible to submit a rolling Biologic License Application (BLA) for apitegromab if relevant criteria are met.
  • We believe the recently announced top-line data from the TOPAZ Phase 2 trial showed the transformative potential of apitegromab for patients with SMA.
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.

2021 Report on the Global Spinal Muscular Atrophy Market - Epidemiology and Patient Flow - ResearchAndMarkets.com

Retrieved on: 
Tuesday, May 11, 2021
Surgery, Health, Physical therapy, Neurological disorders, Branches of biology, Medical specialties, Motor neuron diseases, Neuromuscular Disorders, Gross pathology, Neurogenetic disorders, Muscle atrophy, Atrophy, Draft:Hamano Tsukamoto Syndrome, Spinal muscular atrophies

b'The "Global Spinal Muscular Atrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nThe research report provides Spinal Muscular Atrophy epidemiology, demographics, and patient flow.

Key Points: 
  • b'The "Global Spinal Muscular Atrophy Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nThe research report provides Spinal Muscular Atrophy epidemiology, demographics, and patient flow.
  • The data is presented by Pharma G7 countries including the US, France, Spain, Italy, UK, Japan.\nThe research provides population data to characterize Spinal Muscular Atrophy patients, history of the disease at the population level (Spinal Muscular Atrophy prevalence, Spinal Muscular Atrophy incidence) and at the clinical level (from diagnosis to treated patients).
  • It also helps to identify patient sub-groups (age, gender, sub-groups) to understand targeted population for research and development, commercialization.\nSpinal Muscular Atrophy patient flow: Spinal Muscular Atrophy prevalence, diagnosed, and drug-treated patients\nDemographics: Spinal Muscular Atrophy patients by age group, gender\nThe data from this research will help executives:\nEstablish basis for Spinal Muscular Atrophy market sizing, assessing market potential, and developing drug forecast models\nIdentify Spinal Muscular Atrophy patients segments through age groups, gender, and disease sub-types\nEvaluate Spinal Muscular Atrophy market opportunities, identify target patient population\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210511005653/en/\n'