Channelopathies

Aldeyra Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for ADX-2191 to Treat Retinitis Pigmentosa

Retrieved on: 
Wednesday, August 4, 2021
Biotechnology, FDA, Other Health, Health, General Health, Pharmaceutical, Optical, Other Science, Research, Science, Clinical Trials, Clinical medicine, Medicine, Blindness, Channelopathies, Retinitis pigmentosa, Retinitis, Intravitreal administration, Health

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for ADX-2191 (methotrexate for intravitreal injection) for the treatment of retinitis pigmentosa (RP).

Key Points: 
  • Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for ADX-2191 (methotrexate for intravitreal injection) for the treatment of retinitis pigmentosa (RP).
  • Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology, stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra.
  • "ADX-2191 has now received orphan designations for three distinct clinical indications, highlighting the broad platform potential of ADX-2191 to treat an array of rare retinal disorders.
  • Aldeyra Therapeutics is a biotechnology company developing novel immune-modulating therapies to treat ocular and systemic diseases.

AGTC to Participate at Upcoming Investor Conferences

Retrieved on: 
Monday, August 2, 2021
Vision, Channelopathies, Nervous system, Sensory systems, Blindness, Retinitis pigmentosa, Wedbush Securities, Achromatopsia, Retinitis, Applied Genetic Technologies Corporation

2021 Wedbush PacGrow Healthcare Virtual Conference (August 10-11, 2021)

Key Points: 
  • 2021 Wedbush PacGrow Healthcare Virtual Conference (August 10-11, 2021)
    Ms. Washer will participate in a panel discussion entitled: Only Have Eyes For You - Ophthalmic Gene Therapy at 8:00 a.m.
  • Audio webcasts of the presentations at the Wedbush and H.C. Wainwright conferences can be accessed by visiting http://ir.agtc.com/events-and-presentations .
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

Certilytics, Carewise Health to sponsor Cure CF golf tournament

Retrieved on: 
Thursday, July 22, 2021
Medical specialties, Channelopathies, Cystic fibrosis, Clinician, Cystic Fibrosis Canada, Cystic Fibrosis Trust

LOUISVILLE, Ky., July 22, 2021 /PRNewswire/ -- Certilytics, Inc., and Carewise Health, Inc., are proud to be Diamond Sponsors of the 2021 Cure CF Classic, a golf tournament that will raise money for cystic fibrosis research and care.

Key Points: 
  • LOUISVILLE, Ky., July 22, 2021 /PRNewswire/ -- Certilytics, Inc., and Carewise Health, Inc., are proud to be Diamond Sponsors of the 2021 Cure CF Classic, a golf tournament that will raise money for cystic fibrosis research and care.
  • "The families who are part of Cure CF have seen firsthand the challenges that come with cystic fibrosis," said Joseph Klausing, executive director, Cure CF.
  • Through Certilytics' continued support of Cure CF, we are one day closer to CF standing for Cure Found."
  • Carewise Health, Inc. provides healthcare organizations with Care Management and Payment Integrity solutions that empower clinicians, patients, and health plans to achieve and sustain better health.

Strongbridge Biopharma plc Announces Publication of Long-term Efficacy and Safety Results for KEVEYIS® (dichlorphenamide) for the Treatment of Primary Periodic Paralysis in Muscle & Nerve

Retrieved on: 
Tuesday, July 13, 2021
Rare diseases, Branches of biology, Organ systems, Musculoskeletal disorders, Channelopathies, Electrolyte disturbances, Nephrology, Potassium, Hypokalemic periodic paralysis, Periodic paralysis, Hyperkalemic periodic paralysis, Diclofenamide

The results confirmed that long-term treatment with KEVEYIS is safe and effective for chronic use.

Key Points: 
  • The results confirmed that long-term treatment with KEVEYIS is safe and effective for chronic use.
  • Collectively, these results reinforce the utility of KEVEYIS as an effective option in treating PPP, a life-long rare, hereditary skeletal muscle disorder that leads to debilitating attacks of muscle weakness.
  • KEVEYIS (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.
  • The companys rare neuromuscular franchise includes KEVEYIS (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis.

AGTC to Host Virtual R&D Day on July 22, 2021

Retrieved on: 
Thursday, July 8, 2021
Vision, Channelopathies, Nervous system, Sensory systems, Blindness, Retinitis pigmentosa, Achromatopsia, Applied Genetic Technologies Corporation

We look forward to taking a deeper dive into this data during our R&D Day and discussing preparations for late-stage development of our ACHM and XLRP candidates.

Key Points: 
  • We look forward to taking a deeper dive into this data during our R&D Day and discussing preparations for late-stage development of our ACHM and XLRP candidates.
  • AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases.
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

AGTC Reports 12-Month Data from its Ongoing Phase 1/2 Achromatopsia Clinical Trials Showing Biologic Activity in Patients with Mutations in the ACHM B3 Gene

Retrieved on: 
Thursday, June 24, 2021
Branches of biology, Vision, Biology, Channelopathies, Blindness, Retinitis pigmentosa, Achromatopsia, Clinical trial, Gene therapy, Applied Genetic Technologies Corporation, Retinitis

Based on these data, AGTC intends to advance the ACHM B3 trial to the next stage of clinical development.

Key Points: 
  • Based on these data, AGTC intends to advance the ACHM B3 trial to the next stage of clinical development.
  • The data from the ACHM B3 trial support further clinical investigation of this candidate, and data from additional pediatric patients may support the focused development of the ACHM A3 candidate specifically in younger patients.
  • We are incredibly pleased with these data, which further support AGTC as a leader in the field of ophthalmic gene therapy.
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3).

AGTC to Host Conference Call on June 24 at 8:00 AM ET to Discuss Achromatopsia 12-Month Data in its Ongoing CNGB3 and CNGA3 Phase 1/2 Clinical Trials

Retrieved on: 
Wednesday, June 23, 2021
Vision, Branches of biology, Ion channels, Nervous system, Channelopathies, Cyclic nucleotide gated channel beta 3, Achromatopsia, Cyclic nucleotide-gated channel alpha 3, Retinitis pigmentosa, Retinitis, Applied Genetic Technologies Corporation

AGTC management will host a conference call and webcast with accompanying slides beginning at 8:00 AM Eastern Time on the same date to review the ACHM CNGB3 and CNGA3 data and provide an update on the trials.

Key Points: 
  • AGTC management will host a conference call and webcast with accompanying slides beginning at 8:00 AM Eastern Time on the same date to review the ACHM CNGB3 and CNGA3 data and provide an update on the trials.
  • The live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations .
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

AGTC to Present at American Optometric Association’s Optometry’s Meeting  

Retrieved on: 
Tuesday, June 22, 2021
Branches of biology, Vision, Biology, Channelopathies, Ion channels, Achromatopsia, Agnosía, Visual perception, Visual system, Applied Genetic Technologies Corporation, Cyclic nucleotide gated channel beta 3, Cyclic nucleotide-gated channel alpha 3

Dr. Lin also will also lead three continuing education courses related to gene therapy and IRDs.

Key Points: 
  • Dr. Lin also will also lead three continuing education courses related to gene therapy and IRDs.
  • AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases.
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

Global Retinitis Pigmentosa (Retinitis) Clinical Trials Review - H1, 2021 - ResearchAndMarkets.com

Retrieved on: 
Monday, June 21, 2021
Pharmaceutical, Health, Clinical trials, Health sciences, Health, Blindness, Channelopathies, Retinitis pigmentosa, Clinical trial

The "Retinitis Pigmentosa (Retinitis) - Global Clinical Trials Review, H1, 2021" clinical trials has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Retinitis Pigmentosa (Retinitis) - Global Clinical Trials Review, H1, 2021" clinical trials has been added to ResearchAndMarkets.com's offering.
  • This report provides an overview of Retinitis Pigmentosa (Retinitis) Clinical trials scenario.
  • This report provides top line data relating to the clinical trials on Retinitis Pigmentosa (Retinitis) .
  • Clinical trials are collated from 80+ different clinical trial registries, conferences, journals, news etc across the globe.

Teva Presents New Data at 7th European Academy of Neurology Congress Suggesting Socio-Economic Factors May Contribute to the Significant Under-Diagnosis of Migraine

Retrieved on: 
Monday, June 21, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Migraine, Headaches, Branches of biology, Nervous system, Medicine, Neurological disorders, Channelopathies, Rare diseases, Migraine-associated vertigo, Messoud Ashina, the Epidemiological Study, the ‘Beyond Migraine: The Real You survey, Teva, THE EPIDEMIOLOGICAL STUDY, THE ‘BEYOND MIGRAINE: THE REAL YOU SURVEY, TEVA

A retrospective study of migraine epidemiology in Southern Israel, supported by Teva and presented at the virtual 7th Congress of the European Academy of Neurology (EAN), found extreme variability in the prevalence of diagnosed patients with migraine across the geographic area studied.

Key Points: 
  • A retrospective study of migraine epidemiology in Southern Israel, supported by Teva and presented at the virtual 7th Congress of the European Academy of Neurology (EAN), found extreme variability in the prevalence of diagnosed patients with migraine across the geographic area studied.
  • Migraine is a difficult, challenging neurological disease which does not discriminate dependent on an individuals social status.
  • We believe migraine should be prioritised as a highly prevalent and often under-looked neurological disorder.
  • A 2020 Teva-sponsored research project, Beyond Migraine: The Real You, surveyed over 7,500 people living with the disease in 10 European countries.