Rett syndrome

Acadia Pharmaceuticals Presents New DAYBUE™ (trofinetide) Clinical Data at the 2024 American Academy of Neurology (AAN) Annual Meeting

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水曜日, 4月 17, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, CO, American Academy, Patient, Ageing, Safety, Rett syndrome, Treatment, AAN, Caregiver, Study, Trofinetide, ACAD, Residential care

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that two oral presentations featuring DAYBUE™ (trofinetide) findings in Rett syndrome will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that two oral presentations featuring DAYBUE™ (trofinetide) findings in Rett syndrome will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.
  • The presentations include outcomes from the DAFFODIL™, a Phase 2/3 open-label study evaluating the safety, tolerability and exploratory efficacy of DAYBUE in girls aged two to four living with Rett syndrome, as well as findings from exit interviews with caregivers of individuals living with Rett syndrome who participated in the LAVENDER™, LILAC™, LILAC-2™ and DAFFODIL studies.
  • Oral Presentation 003/Abstract 3540: Trofinetide for the Treatment of Girls Aged Two to Four Years with Rett Syndrome: Final Results from the Open-label DAFFODIL Study, Wednesday, April 17 at 3:54pm MT
    Oral Presentation 008/Abstract 2877: Assessing Experiences with Trofinetide for Rett Syndrome: Interviews with Caregivers of Patients in Open-label Studies, Wednesday, April 17 at 4:54pm MT

May 13, 2024 Deadline: Contact The Gross Law Firm to Join Class Action Suit Against AVXL

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金曜日, 4月 26, 2024
MCI, News, Radar, NEXT, Rett syndrome, Alzheimer's disease, Patient, Clinical trial, Fraud, Security (finance)

NEW YORK, April 26, 2024 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Anavex Life Sciences Corporation (NASDAQ: AVXL).

Key Points: 
  • NEW YORK, April 26, 2024 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Anavex Life Sciences Corporation (NASDAQ: AVXL).
  • Shareholders who purchased shares of AVXL during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment.
  • The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices.
  • The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship.

Rett Syndrome Research Trust Partners with Vivalink to Shift Symptom Assessment Paradigms in Rett Syndrome

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火曜日, 4月 23, 2024
RSRT, Neurological disorder, Health, Rett syndrome, Respiratory rate, Partnership, Research, ECG, Heart rate, Speech, Therapy, Patient, Data collection, Caregiver, Posture, Drug development, Biomarker, Genetic disorder, Sleep, Frasier, Dysautonomia, Medical device

CAMPBELL, Calif., April 23, 2024 /PRNewswire/ -- Vivalink, a leading provider of digital healthcare solutions, announces its collaboration with the Rett Syndrome Research Trust (RSRT) to advance research into Rett syndrome, a rare neurological disorder. As part of this partnership, Vivalink is providing its Multi-Vital ECG wearable technology and data services for heart rate and oxygen saturation monitoring to support RSRT's new VIBRANT study, aimed at assessing autonomic dysfunction in individuals with Rett syndrome.

Key Points: 
  • CAMPBELL, Calif., April 23, 2024 /PRNewswire/ -- Vivalink , a leading provider of digital healthcare solutions, announces its collaboration with the Rett Syndrome Research Trust (RSRT) to advance research into Rett syndrome, a rare neurological disorder.
  • Vivalink's advanced wearable devices provide an important opportunity to expedite drug development in the pursuit of a cure for Rett Syndrome.
  • "We are excited to partner with the Rett Syndrome Research Trust in their pioneering study," said Aaron Timm, Chief Commercial Officer at Vivalink.
  • "We hope to empower researchers with the tools they need to make meaningful advancements in understanding and treating Rett syndrome."

MAKING A SPLASH: APEX SOCIAL CELEBRATES CAREGIVERS WITH SURF, SAND, AND SERVICE

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火曜日, 4月 16, 2024
Knowledge, Education, Person-centered therapy, Making a Splash, Rocky, Woman, State, Building Bridges, Rett syndrome, Love, Pacific Ocean, Nursing, Family, Professional, Child care, Beauty, SAN

Apex Social Group provides families with developmental live-in childcare and cultural exchange.

Key Points: 
  • Apex Social Group provides families with developmental live-in childcare and cultural exchange.
  • Amanda Spagnoletti, a host mom from New Jersey, is just one of many who sing the praises of her Care Professionals.
  • "Creating this event is a true labor of love," says Susan Asay, Founder of Apex Social Group and long-time San Clemente resident.
  • According to Sarah Kodinsky, CEO at Apex Social, "The excitement for the event is palpable year after year.

Rett Syndrome Research Trust Launches the MECP2 Editing Consortium With Goal to Advance Programs to Clinical Trials

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水曜日, 4月 10, 2024
FDA, Massachusetts Institute of Technology, RSRT, Harvard University, University, Rett syndrome, Gene editing, RNA, MD, Doctor of Philosophy, Research, DNA, Gene, Clinical trial, Pharmaceutical industry

TRUMBULL, Conn. , April 10, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium. Mutations in MECP2 cause Rett syndrome, a debilitating neurological disorder that causes a lifetime of severe disabilities. The consortium comprises six distinguished labs, including Adrian Bird, PhD and Jacky Guy, PhD from the University of Edinburgh, Erik Sontheimer, PhD, Jonathan Watts, PhD and Scot Wolfe, PhD from University of Massachusetts Chan Medical School, Guoping Feng, PhD and Chenjie Shen, PhD from Massachusetts Institute of Technology, and Peter Beal, PhD from the University of California, Davis. The editing consortium, supported with a $7 million investment from RSRT, is inspired by the prior success of RSRT's Gene Therapy Consortium which laid the foundation for the current gene therapy trials. RSRT is the largest funder of Rett research with more than $77 million awarded since 2008 to advancing cures.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium.
  • TRUMBULL, Conn. , April 10, 2024 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium.
  • The consortium's inaugural meeting earlier this year set an ambitious agenda to advance editing programs to clinical trials.
  • I'm very much looking forward to working with everyone in the consortium as the programs advance toward the clinic."

Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites Treatment of Rett syndrome, 12/01/2024 Positive

Retrieved on: 
木曜日, 4月 18, 2024
MECP2, Patient, Committee, EMA, IRIS, Rett, Rett syndrome, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for treatment of the Rett syndrome on 12 January 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Bragar Eagel & Squire, P.C. Reminds Investors That Class Action Lawsuits Have Been Filed Against Ventyx, and Anavex and Encourages Investors to Contact the Firm

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火曜日, 4月 9, 2024
Psoriasis, Form, Patient, Defendant, News, IPO, Crohn's disease, Security (finance), Inflammation, Rett syndrome, Prospectus, Complaint, P.C, Clinical trial, MCI, Radar, File, Register, Pharmaceutical industry

The Company's lead clinical product candidate is VTX958, a selective allosteric tyrosine kinase type 2 inhibitor for psoriasis, psoriatic arthritis, and Crohn's disease.

Key Points: 
  • The Company's lead clinical product candidate is VTX958, a selective allosteric tyrosine kinase type 2 inhibitor for psoriasis, psoriatic arthritis, and Crohn's disease.
  • In addition, throughout the Class Period, Defendants made materially false and misleading statements regarding the Company's business, operations, and prospects.
  • On November 6, 2023, during after-market hours, Ventyx issued a press release announcing results from the Phase 2 SERENITY Trial.
  • For more information on the Anavex class action go to: https://bespc.com/cases/AVXL

SHAREHOLDER ALERT: Anavex Life Sciences Sued for Securities Law Violations; Investors Should Contact Block & Leviton for More Information

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金曜日, 4月 5, 2024
Patient, Certification, Block & Leviton, Rett syndrome, Clinical trial, Cryptocurrency

BOSTON, April 05, 2024 (GLOBE NEWSWIRE) -- A class action lawsuit has been filed against Anavex Life Sciences Corp. (NASDAQ: AVXL) for securities law violations.

Key Points: 
  • BOSTON, April 05, 2024 (GLOBE NEWSWIRE) -- A class action lawsuit has been filed against Anavex Life Sciences Corp. (NASDAQ: AVXL) for securities law violations.
  • Investors who purchased shares and have lost money are encouraged to contact the firm to learn more about how they might recover those losses.
  • The study failed to show statistical significance towards its primary endpoints, causing shares of Anavex Life Sciences Corp. stock to drop approximately 35%.
  • Block & Leviton is widely regarded as one of the leading securities class action firms in the country.

Robbins LLP Reminds Investors to Seek Counsel in the Class Action Against Anavex Life Sciences Corporation (AVXL)

Retrieved on: 
金曜日, 4月 5, 2024
Rett, Excellence, Central nervous system, Patient, SAN, CNS, Rett syndrome, Complaint, News, LLP, Person, Pharmaceutical industry

SAN DIEGO, April 05, 2024 (GLOBE NEWSWIRE) -- Robbins LLP reminds investors that a shareholder filed a class action on behalf of all persons who purchased or otherwise acquired Anavex Life Sciences Corporation (NASDAQ: AVXL) stock between February 1, 2022 and January 1,2024.

Key Points: 
  • SAN DIEGO, April 05, 2024 (GLOBE NEWSWIRE) -- Robbins LLP reminds investors that a shareholder filed a class action on behalf of all persons who purchased or otherwise acquired Anavex Life Sciences Corporation (NASDAQ: AVXL) stock between February 1, 2022 and January 1,2024.
  • For more information, submit a form , email attorney Aaron Dumas, Jr., or give us a call at (800) 350-6003.
  • Anavex sponsored the “Excellence” Phase II/Phase III study to investigate blarcamesine as a treatment for pediatric Rett syndrome patients.
  • What Now: You may be eligible to participate in the class action against Anavex Life Sciences Corporation.

Bragar Eagel & Squire, P.C. Reminds Investors That Class Action Lawsuits Have Been Filed Against Hut 8, Ventyx, and Anavex and Encourages Investors to Contact the Firm

Retrieved on: 
金曜日, 4月 5, 2024
Bitcoin, Research, Bankruptcy, Psoriasis, Register, Form, Patient, Defendant, News, IPO, Crohn's disease, Security (finance), Data mining, Inflammation, Rett syndrome, Prospectus, Complaint, Hut 8, P.C, Clinical trial, MCI, Radar, File, Mergers and acquisitions, Cryptocurrency

In November 2023, Hut 8 formed following the merger of Hut 8 Mining Corp. (“Legacy Hut”) and U.S. Data Mining Group, Inc. d/b/a US Bitcoin Corp. (“USBTC”) (the “Merger”).

Key Points: 
  • In November 2023, Hut 8 formed following the merger of Hut 8 Mining Corp. (“Legacy Hut”) and U.S. Data Mining Group, Inc. d/b/a US Bitcoin Corp. (“USBTC”) (the “Merger”).
  • USBTC held a 50% interest in a joint venture bitcoin mining facility, located in King Mountain, Texas (the “King Mountain JV”), which was acquired in the Merger.
  • In addition, throughout the Class Period, Defendants made materially false and misleading statements regarding the Company's business, operations, and prospects.
  • For more information on the Anavex class action go to: https://bespc.com/cases/AVXL