FXS

Spinogenix Announces U.S. FDA Orphan Drug Designation Granted to SPG601 for the Treatment of Fragile X Syndrome

Retrieved on: 
Montag, Mai 20, 2024
ODD, Attention deficit, Intellectual disability, Patient, Fragile X syndrome, FMR1, BK, Food, Aggression, FXS, FDA, Doctor of Philosophy, Spectrum, Anxiety, SAN, Woman, Synapse, Therapy, Pharmaceutical industry

SAN DIEGO, May 20, 2024 (GLOBE NEWSWIRE) -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SPG601 for the treatment of Fragile X Syndrome (FXS).

Key Points: 
  • SAN DIEGO, May 20, 2024 (GLOBE NEWSWIRE) -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SPG601 for the treatment of Fragile X Syndrome (FXS).
  • “Receiving ODD for SPG601 underscores the high unmet need for new treatment options for individuals with FXS,” said Craig Erickson, M.D., Spinogenix Chief Medical Advisor.
  • “Despite the challenges individuals living with FXS endure, there are currently no FDA-approved therapies available for this condition.
  • We believe people living with Fragile X need a therapeutic option that works at the synaptic level to address their wide range of disabling symptoms.

Spinogenix Announces U.S. FDA Approval of its Investigational New Drug Application for its Phase 2a Clinical Trial of SPG601 for Fragile X Syndrome

Retrieved on: 
Montag, April 15, 2024
FDA, BK, Hope, Fragile X syndrome, Spectrum, Woman, Food, FMR1, FXS, IND, Aggression, Disease, Human, Therapy, Patient, Intellectual disability, Synapse, Anxiety, SAN, Investigational New Drug, Attention deficit, Pharmaceutical industry

SPG601 works by restoring synaptic function to address core symptoms of FXS.

Key Points: 
  • SPG601 works by restoring synaptic function to address core symptoms of FXS.
  • The Phase 2a trial will evaluate the neurophysiological and clinical effects of single-dose SPG601 and placebo in adult men with FXS.
  • SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to restore synaptic function.
  • We look forward to the first study of a BK channel modulator in humans with Fragile X and taking the first step to evaluate this important drug mechanism in Fragile X Syndrome.”

TeleRare Health™ Announces Launch of National Virtual Clinic for Rare Disease Patients

Retrieved on: 
Dienstag, April 30, 2024
Wilson's disease, Bangladesh Technical Education Board, Genomics, Kidney, SCID, Diagnosis, Safety, NIH, AI, Heart, Adrenoleukodystrophy, MD, Partnership, Children's National Hospital, DMD, FXS, Research, Human, Genetic testing, Disease, Patient, ALD, Pediatrics, Krabbe disease, Duchenne muscular dystrophy, PD, GMG, Fragile X syndrome, Pharmaceutical industry

MINNEAPOLIS, April 30, 2024 /PRNewswire/ -- 8:00a (CET) April 30, 2024 - TeleRare Health™ launched today, a virtual clinic for Rare Disease patients and their families.

Key Points: 
  • MINNEAPOLIS, April 30, 2024 /PRNewswire/ -- 8:00a (CET) April 30, 2024 - TeleRare Health™ launched today, a virtual clinic for Rare Disease patients and their families.
  • TeleRare Health aims to be the premier provider of virtual care for rare and genomic disease nationwide, envisioning a world where all patients have access to cutting edge diagnostics, treatments, and specialists.
  • "Significant breakthroughs have occurred in the management of rare disease," said Alex Katz, MD, medical geneticist and Chief Medical Officer of TeleRare Health.
  • We're overdue for a virtual clinic for the millions of Americans with rare disease conditions, and TeleRare Health's launch is cause for optimism.

Rgenta Therapeutics to Present at the 19th Annual Huntington's Disease Therapeutics Conference

Retrieved on: 
Mittwoch, Februar 21, 2024
Traffic barrier, MLH1, PMS2, Huntington's disease, GWAS, FXS, Conference, DM1, MSH3, PMS1, RNA, Therapy, FAN1, Gene, FRDA, CSO, Fragile X syndrome, Friedreich's ataxia, DNA, Vaccine

CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.
  • As part of the annual conference, Travis Wager, Ph.D., President & CSO of Rgenta will participate in the Targeting the DNA repair machinery to modulate somatic instability session on Tuesday, February 27, 2024 at 2:00 PM PT.
  • To date, there has been a lack of successful strategies for directly targeting the PMS1 protein with small molecule inhibitors.
  • For more information about the event, please visit the conference website .

Flash News: OKX to List FXS and LQTY Perpetual Swaps, Enables Margin Trading and Simple Earn for the Tokens

Retrieved on: 
Freitag, Januar 12, 2024
API, FXS, UTC, OKX, Token, Cryptocurrency

OKX to List FXS and LQTY Perpetual Swaps, Enables Margin Trading and Simple Earn for the Tokens

Key Points: 
  • OKX to List FXS and LQTY Perpetual Swaps, Enables Margin Trading and Simple Earn for the Tokens
    OKX will today list two tokens on its perpetual futures market: FXS and LQTY.
  • These additions will enable users to trade FXS/USDT and LQTY/USDT with up to 20x leverage from 8:00 AM (UTC) and 10:00 AM (UTC).
  • OKX today also enabled margin trading and Simple Earn for FXS and LQTY at 7:30 am (UTC) and 9:30 am (UTC), respectively.
  • This announcement follows the listing of NMR/USDT and JOE/USDT on OKX's perpetual futures market on January 11.

T1 E1 Network Test Solution Capabilities

Retrieved on: 
Donnerstag, November 2, 2023
CAS, DCE, Linux, PC, WCS, DTE, GSM, Analyser, Command-line interface, SSH, FXS, GL, Telephone, Python, Unix, WLC, PPP, Maintenance, USB, Frame Relay, SS7, Microsoft Windows, Telecom, PowerShell, ISDN, SS1, FXO, Probe, Collection, HDLC, Automation, Data analysis, Communication, Console, Emulation, Mobile phone, Video game, Internet service provider, Computer data storage, Computer network

“GL's tProbe™ is a test and measurement device for T1 E1 networks.

Key Points: 
  • “GL's tProbe™ is a test and measurement device for T1 E1 networks.
  • GL's Windows Client/Server (WCS) software allows T1 E1 analysis and emulation cards with remote operation, automation, and multi-site connectivity capabilities.
  • It sends commands to T1 E1 WCS servers and shows the responses in Console, PowerShell, or Terminal Windows.
  • Available with Dual T1 or E1, FXO, FXS, DTE and DCE interfaces
    Bit Error Rate Testing over all timeslots.

Harmony Biosciences Reports Strong Third Quarter 2023 Financial Results

Retrieved on: 
Dienstag, Oktober 31, 2023
IH, DM1, FDA, Research, Acquisition, Prader–Willi syndrome, Continue, Pivotal, LCA, Fragile X syndrome, Narcolepsy, FXS, Share repurchase, Conference, GAAP, Idiopathic hypersomnia, Bank statement, Development, Common stock, Commercialization, Growth, DSM-IV codes, Research and development, Benchmarking, PWS, Total, Myotonic dystrophy, Patient, Administration, Marketing, Shanda, Video game, Pharmaceutical industry, Fine chemical, Car rental, Silviculture, Cryptocurrency, Bank, Jean-François Zygel, Webcast, Harmony

ET

Key Points: 
  • ET
    PLYMOUTH MEETING, Pa., Oct. 31, 2023 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. (“Harmony” or the “Company”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today reported financial results and business updates for the quarter ended September 30, 2023.
  • The average number of patients on WAKIX increased by approximately 350 sequentially to approximately 5,800 for the quarter ended September 30, 2023.
  • Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release.
  • ET
    We are hosting our third quarter 2023 financial results conference call and webcast today, beginning at 8:30 a.m. Eastern Time.

New York State Laboratory Approved for Genetic Testing Using Saliva

Retrieved on: 
Dienstag, Oktober 17, 2023
Diagnosis, American Academy of Pediatrics, FXTAS, Fragile X syndrome, Fragile X-associated tremor/ataxia syndrome, FXS, Saliva, FMR1, Laboratory, Intellectual disability, NYSDOH, Adolescence, Fragile X-associated primary ovarian insufficiency, Genetic testing, Child, Blood, New York State Department of Health, EVP, Spectrum, Physician, Vaccine, Quadrant

SYRACUSE, N.Y., Oct. 17, 2023 /PRNewswire/ -- Quadrant Laboratories announced today that its Syracuse-based lab has been approved by the New York State Department of Health for genetic testing. This is in conjunction with the NYSDOH approval of Quadrant's Fragile X Syndrome (FXS) test, which is now available for physicians to order in all 50 states. Quadrant's FXS test uses a saliva swab as a primary collection, rather than a blood draw, and can be collected in the patient's home before getting shipped back to be tested in Quadrant's CLIA/CLEP certified clinical lab.

Key Points: 
  • SYRACUSE, N.Y., Oct. 17, 2023 /PRNewswire/ -- Quadrant Laboratories announced today that its Syracuse-based lab has been approved by the New York State Department of Health for genetic testing.
  • Dr. Funda Suer, EVP of Clinical Diagnostics and Clinical Laboratory Director for Quadrant Laboratories, said, "In both the pediatric and adolescent populations, we found it was much easier to collect saliva versus blood.
  • This genetic condition  is caused by changes in the FMR1 gene, which is responsible for creating a protein crucial in brain development.
  • Following the discovery of the association between the FMR1 gene and FXS, gene-specific testing has made identifying the condition significantly more accurate.

Zynerba Pharmaceuticals Stockholders Reminded to Tender Shares Before 5:00 PM New York City Time, on Tuesday October 10, 2023

Retrieved on: 
Freitag, September 29, 2023
Documentation, Agreement, Letter, Plan, CVR, Failure, Completeness, Tripura Merger Agreement, TD Ameritrade, Biotechnology, Stock dilution, Food and Drug Administration, Depositary, Bangladesh Securities and Exchange Commission, Schedule, Harmony, FXS, Assistance, Letter of transmittal, Fragile X syndrome, Continue, ...Continued, Less Commonly Taught Languages, Security (finance), FDA, Acquisition, Food, Personal Information Agent, Action Replay, Pharmaceutical industry, Cryptocurrency, Holding company, Sales, Mail order, Reinsurance, Money transmitter, Jean-François Zygel

The Offer was initially scheduled to expire at 5:00 p.m. New York City time on September 26, 2023.

Key Points: 
  • The Offer was initially scheduled to expire at 5:00 p.m. New York City time on September 26, 2023.
  • On September 27, 2023, Harmony announced an extension of the Offer until 5:00 p.m., New York City time, on Tuesday, October 10, 2023, to allow for additional shares to be tendered by stockholders.
  • I am reaching out to remind you to tender your shares prior to the expiration of the Offer on October 10, 2023.
  • The Zynerba Board unanimously recommends that Zynerba stockholders tender their shares pursuant to the Offer.

Zatolmilast, an Investigational Treatment for Fragile X Syndrome, Receives Rare Pediatric Disease Designation from the U.S. FDA

Retrieved on: 
Mittwoch, September 27, 2023
Research, Mental Health, Neurology, FDA, Clinical Trials, General Health, Pharmaceutical, Health, Science, Other Science, Tetra, Cognition, Camp, Neuron, Life, AMP, Vocabulary, Head, Medicine, Bangladesh Technical Education Board, Disorder, Intellectual disability, Vomiting, FXS, Fragile X syndrome, Genetic disorder, Lower respiratory tract infection, JD, Spectrum, RPD, AICA ribonucleotide, Adolescence, Peabody Picture Vocabulary Test, Orphan, Oral, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry, Shionogi

FDA grants Rare Pediatric Disease Designation (RPD) for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States.

Key Points: 
  • FDA grants Rare Pediatric Disease Designation (RPD) for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States.
  • The RPD designation allows Tetra to request a priority review voucher from FDA which, if granted, may be used for a subsequent human drug application.
  • Zatolmilast was also awarded Orphan Drug Designation by the FDA in 2018.
  • “We are committed to advancing a potential medicine for people with Fragile X syndrome, a rare genetic disorder which affects all aspects of life for individuals and families.