Neurological disorders

AcuraStem Awarded $1M DOD Grant to Advance AS-202 Drug Development Candidate for ALS

Retrieved on: 
Tuesday, May 25, 2021
Branches of biology, Neurological disorders, Neurodegenerative disorders, Amyotrophic lateral sclerosis, Neurodegeneration

Through CDMRP's award mechanisms and funding recommendations, the ALSRP highlights innovative and impactful research targeting the development of new therapeutics for ALS.

Key Points: 
  • Through CDMRP's award mechanisms and funding recommendations, the ALSRP highlights innovative and impactful research targeting the development of new therapeutics for ALS.
  • Because the platform includes neurons from patients with sporadic ALS, AcuraStem is able to identify novel therapeutic approaches that could be disease-modifying for all ALS patients.
  • AcuraStem is a patient-based drug discovery platform company developing novel drug programs for neurodegenerative diseases, starting with ALS.
  • All of AcuraStem's drug discovery is done using neurons derived from patients and modeled on its proprietary iNeuroRx technology platform.

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.

Rett Syndrome Research Trust Establishes Translational Medicine Advisory Board

Retrieved on: 
Thursday, May 20, 2021
Neurological disorders, Branches of biology, Autism, Psychiatric diagnosis, Rett syndrome, Clinical medicine, Rett, Translational medicine, CDKL5

b'TRUMBULL, Conn., May 20, 2021 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust has established a Translational Medicine Advisory Board to provide new strategic direction for its CURE 360 agenda and open new avenues of innovative research that can be applied to Rett Syndrome.

Key Points: 
  • b'TRUMBULL, Conn., May 20, 2021 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust has established a Translational Medicine Advisory Board to provide new strategic direction for its CURE 360 agenda and open new avenues of innovative research that can be applied to Rett Syndrome.
  • "The Translational Medicine Advisory Board will help us focus our resources on the most promising areas of research, facilitate introductions to scientists and biopharma companies working on impactful new therapeutic approaches and technologies, and bring us closer to a cure.
  • They share our passion and urgency and are dedicated to working with us to advance the development of therapeutics targeting the root cause of Rett Syndrome," said Dr. Randy Carpenter, Chief Medical Officer of the Rett Syndrome Research Trust.\nMembers of the new advisory board:\nSteven E. Hyman, M.D .
  • CURE 360 also ensures that the research incubated at the Rett Syndrome Research Trust moves into biopharma for advancement to clinical trials.\nThanks to the fundraising and support of affected families and their networks, the Rett Syndrome Research Trust can pursue this research strategically and aggressively.\n"We look forward to working with and tapping into the knowledge of our Translational Medicine Advisory Board members, and are certain that with their expertise, we can accelerate our mission.

GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome

Retrieved on: 
Wednesday, May 19, 2021
Branches of biology, Learning disabilities, Neurological disorders, Clinical medicine, Health, Genodermatoses, UBE3A, Angelman syndrome, Chromosome 15, Ube3a-ATS

Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety.

Key Points: 
  • Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety.
  • In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression.
  • While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently.
  • GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research.

Zogenix Launches Dravet Syndrome Community Photo Sharing Charitable Campaign and Contest to Benefit the Dravet Syndrome Foundation and the Epilepsy Foundation

Retrieved on: 
Wednesday, May 19, 2021
Neurological disorders, Drugs, Health, Epilepsy, Dravet syndrome, Orphan drugs, GABAA receptor positive allosteric modulators, Fenfluramine, Phenobarbital

b'EMERYVILLE, Calif., May 19, 2021 /PRNewswire/ --Zogenix, a global biopharmaceutical company developing rare disease therapies, today launched the FINTEPLA Photo Diary, a community sharing, charitable campaign and contest.

Key Points: 
  • b'EMERYVILLE, Calif., May 19, 2021 /PRNewswire/ --Zogenix, a global biopharmaceutical company developing rare disease therapies, today launched the FINTEPLA Photo Diary, a community sharing, charitable campaign and contest.
  • To participate in the charitable campaign and contest, Zogenix is asking the Dravet syndrome community to share photos of their everyday moments in life with fewer seizures on FINTEPLA (fenfluramine) from now through June 9, 2021.
  • Please read Important Safety Information, including Boxed Warning below.\n"We are constantly inspired by the Dravet community and members\' support for one another.
  • "The FINTEPLA Photo Diary campaign and contest is an opportunity for us to share the hope that we have in between seizures.

ALS Association Announces Goal of Making ALS Livable by 2030

Retrieved on: 
Tuesday, May 18, 2021
Neurological disorders, Neurodegenerative disorders, Nervous system, Amyotrophic lateral sclerosis, ALS Association, Als

b'WASHINGTON, May 18, 2021 /PRNewswire/ -- The ALS Association today announced a goal of making ALS a livable disease by 2030.

Key Points: 
  • b'WASHINGTON, May 18, 2021 /PRNewswire/ -- The ALS Association today announced a goal of making ALS a livable disease by 2030.
  • "Making ALS livable also means improving the care we are delivering and thinking about how to prevent ALS and its associated harms, which will help people with ALS and their loved ones.
  • The ALS Association builds hope and enhances quality of life while urgently searching for new treatments and a cure.
  • For more information about The ALS Association, visit our website at www.als.org .\nView original content to download multimedia: http://www.prnewswire.com/news-releases/als-association-announces-goal-o...\n'

Utah PTA Passes Historic Resolution Bringing Migraine and Headache Disease Education into the Classroom

Retrieved on: 
Tuesday, May 18, 2021
Migraine, Medicine, Symptoms and signs, Branches of biology, Headaches, Neurological disorders, Pain management, Headache, Cluster headache

b'SANTA MONICA, Calif., May 18, 2021 /PRNewswire/ --The Utah PTA recently passed an important resolution to bring migraine and headache education into the classroom statewide, while also providing much needed resources for students, parents, administrators, educators, and nurses.

Key Points: 
  • b'SANTA MONICA, Calif., May 18, 2021 /PRNewswire/ --The Utah PTA recently passed an important resolution to bring migraine and headache education into the classroom statewide, while also providing much needed resources for students, parents, administrators, educators, and nurses.
  • There are over 40 million people in the United States with migraine disease, and just overhalf have been diagnosed.
  • Also, there are 400,000 people with cluster headache, recognized as the most painful disease a person can have.
  • The campaign includesa powerful video, Growing up with Migraine , that showcaseswhy CHAMP\'s Migraine at School program is so important for migraine education through stories shared by kids and adults sharing what it is like to grow up with migraine disease.

Loyal Source Raises Over $160,000 for Central Florida Nonprofit During 14th Annual Derby Day Event

Retrieved on: 
Monday, May 17, 2021
Psychiatric diagnosis, Branches of biology, Abnormal psychology, Psychiatry, Autism, Learning disabilities, Neurological disorders, Pervasive developmental disorders, Autism spectrum

Despite hurdles from the ongoing pandemic, this is the largest donation to date stemming from this annual event.\nPALS\xe2\x80\x99s mission is to create opportunities for individuals of all ages with autism spectrum disorders (ASD) by providing state-of-the-art social inclusive community programs in Central Florida.

Key Points: 
  • Despite hurdles from the ongoing pandemic, this is the largest donation to date stemming from this annual event.\nPALS\xe2\x80\x99s mission is to create opportunities for individuals of all ages with autism spectrum disorders (ASD) by providing state-of-the-art social inclusive community programs in Central Florida.
  • \xe2\x80\x9cThis generous donation will go directly to support our work with individuals and families living with autism and related disorders throughout Central Florida.\xe2\x80\x9d\nLoyal Source is committed to supporting the Central Florida community through philanthropic initiatives.
  • With a focus in government healthcare, technical and support services, engineering, and travel healthcare, Loyal Source provides exceptional custom solutions to both private enterprise and government agencies.
  • For more information about Loyal Source, visit https://loyalsource.com/ .\n'

Neuropore Therapies Receives $20 Million (USD) Milestone Payment Under Collaboration and License Agreement with UCB

Retrieved on: 
Monday, May 17, 2021
Biotechnology, Pharmaceutical, Health, Other Health, Branches of biology, Peripheral membrane proteins, Neurological disorders, Alpha-synuclein, Lewy body dementia, Synuclein, Neurodegeneration, UCB

b'Neuropore Therapies, Inc. announced today that it has earned a milestone payment related to its collaboration with UCB and the evaluation of UCB0599 for the treatment of Parkinson\xe2\x80\x99s Disease.

Key Points: 
  • b'Neuropore Therapies, Inc. announced today that it has earned a milestone payment related to its collaboration with UCB and the evaluation of UCB0599 for the treatment of Parkinson\xe2\x80\x99s Disease.
  • UCB0599 is an orally administered small molecule alpha-synuclein misfolding inhibitor arising from a Collaboration and License Agreement entered into between Neuropore Therapies, Inc and UCB Biopharma SRL on December 31, 2014.
  • We believe that inhibition of alpha-synuclein misfolding and oligomerization with an orally active, brain penetrating, small molecule represents a promising therapeutic approach.
  • As a result of this latest milestone achievement, Neuropore Therapies has cumulatively received $63 million (USD) of the potential $460 million (USD) in total milestones available through its collaboration with UCB.\nParkinson\xe2\x80\x99s disease (PD) is the second most common neurodegenerative disorder after Alzheimer\xe2\x80\x99s disease.

Gain Therapeutics to Participate in Upcoming Virtual Investor Conferences in May

Retrieved on: 
Monday, May 17, 2021
Articles, Branches of biology, Business, Protein folding, Neurological disorders, Protein structure, Amyloidosis, Pathology, Proteopathy, Forward-looking statement, Allosteric regulation, Entrepreneurship

By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding.

Key Points: 
  • By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding.
  • Gain was established in 2017 with the support of its founders and institutional investors.
  • Such statements include, but are not limited to, statements regarding the market opportunity for Gain\xe2\x80\x99s product candidates; and the business strategies and development plans of Gain.
  • Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made.