Usher syndrome

Nacuity Pharmaceuticals Announces Expansion of its Business Advisory Board with Appointment of Rare Disease Advocate Daniel Feller

Retrieved on: 
Wednesday, February 14, 2024

FORT WORTH, Texas, Feb. 14, 2024 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other ocular diseases caused by oxidative stress, today announced the expansion of its business advisory board with the appointment of Daniel Feller.

Key Points: 
  • FORT WORTH, Texas, Feb. 14, 2024 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other ocular diseases caused by oxidative stress, today announced the expansion of its business advisory board with the appointment of Daniel Feller.
  • Mr. Feller founded Talisman Licensing, a brand building agency in Melbourne, Australia, and has served as its CEO since the Company’s inception in 2007.
  • Mrs. Feller also co-founded and currently serves as the director of UsherKids Australia, an Australian-based support network for families with children diagnosed with Usher syndrome.
  • “Nacuity’s oxidative stress therapies have broad potential to treat a variety of diseases, including retinitis pigmentosa associated with Usher syndrome.

Foundation Fighting Blindness Hosting Webinar for Eye Care Professionals: Best Clinical Practices for Patients with Inherited Retinal Diseases

Retrieved on: 
Tuesday, February 13, 2024

COLUMBIA, Md., Feb. 13, 2024 /PRNewswire/ -- The Foundation Fighting Blindness, the driving force in the global development of treatments and cures for blinding diseases, will host a webinar for eye care professionals on the best practices for managing their patients with inherited retinal diseases (IRDs), including retinitis pigmentosa, Usher syndrome, Stargardt disease, and Leber congenital amaurosis. This webinar will be a part of the March Envisioning a Path to Hope initiative, raising awareness among eye care professionals for the valuable resources the Foundation provides IRD patients and caregivers.

Key Points: 
  • This webinar will be a part of the March Envisioning a Path to Hope initiative, raising awareness among eye care professionals for the valuable resources the Foundation provides IRD patients and caregivers.
  • "With nearly 50 clinical trials underway for emerging IRD therapies and no-cost genetic testing available for patients, eye care professionals can do so much to help IRD patients on their challenging journeys," says Michelle Glaze, director of professional outreach, Foundation Fighting Blindness.
  • "We are delighted to host this informative webinar to empower eye doctors to communicate hope and a path forward for their IRD patients and families."
  • She treats patients in the clinical departments of Acquired Brain Injury and Low Vision Rehabilitation, where she also conducts genetic testing for inherited eye conditions.

Usher Syndrome Society Commits Another $500,000 for New Round of Usher Syndrome Research Grants

Retrieved on: 
Thursday, July 20, 2023

About the Usher Syndrome SocietyThe USH Society is a non--profit created because of an urgent need to save the sight and hearing of those living with Usher syndrome. Realizing that the two most important ways to accelerate research are educating the public and raising research funds, the USH Society began using photojournalism, film, and educational events to bring Usher syndrome to the forefront of rare diseases. The Usher Syndrome Society is a registered 501(c)3. Visit www.UsherSyndromeSociety.org for more information.

Key Points: 
  • Funded projects will help drive collaborative ear-­‐and-­‐eye Usher syndrome research focused on the discovery pipeline including identification of therapeutic strategies and development of novel therapeutic agents.
  • NEEDHAM, Mass., July 20, 2023 /PRNewswire/ -- The Usher Syndrome Society (USH Society), a non-­‐ profit that uses storytelling through the arts, educational events, and collaboration to raise public awareness and funds for research to find treatments and a cure for Usher syndrome (USH) announces a request for applications for Usher syndrome research grants.
  • The "Usher Syndrome Society Translational Research Grants" are intended to support translational research on Usher syndrome in either Preclinical Research and/or Mechanism-­‐based Therapeutic Development.
  • The USH Society is committed to supporting Usher syndrome research at labs with promising work and specific funding needs that accelerate Usher syndrome research towards treatments and a cure.

Nacuity Pharmaceuticals Achieves Target Enrollment for Phase 1/2 Clinical Trial of NPI-001 for the Treatment of Retinitis Pigmentosa Associated with Usher Syndrome

Retrieved on: 
Wednesday, May 31, 2023

FORT WORTH, Texas, May 31, 2023 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that target enrollment of 48 patients has been reached in the SLO-RP Phase 1/2 clinical trial of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH).

Key Points: 
  • “We are pleased to reach our target enrollment, an important step toward our goal to realize the potential of NPI-001 for patients affected by RP,” said Halden Conner, Chairman, CEO and Co-Founder of Nacuity Pharmaceuticals.
  • “We extend our gratitude to the principal investigators, Usher patient advocacy organizations and clinical sites who supported our recruitment efforts, working through challenges including the early days of a global pandemic.
  • The trial enrolled 48 male and female patients, ages 18 years and older, at its four trial sites in Australia.
  • The protocol has been amended to allow continued treatment beyond two years for interested participants while the trial is ongoing.

Emmy Award-Winning NBC News Journalist Peter Alexander to Host National Organization for Rare Disorders' (NORD) 40th Anniversary Celebration and 2023 Rare Impact Awards

Retrieved on: 
Friday, March 31, 2023

, March 31, 2023 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) announced today that Emmy Award-winning journalist and NBC News Chief White House correspondent, Peter Alexander, will host the 2023 Rare Impact Awards and NORD's 40th Anniversary celebration, Thursday, May 4, 2023 at the Smithsonian National Portrait Gallery in Washington, D.C. Tickets to this black-tie fundraising event are available at rareimpact.org .

Key Points: 
  • , March 31, 2023 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) announced today that Emmy Award-winning journalist and NBC News Chief White House correspondent, Peter Alexander, will host the 2023 Rare Impact Awards and NORD's 40th Anniversary celebration, Thursday, May 4, 2023 at the Smithsonian National Portrait Gallery in Washington, D.C. Tickets to this black-tie fundraising event are available at rareimpact.org .
  • NBC News Chief White House correspondent, Peter Alexander, will host the National Organization for Rare Disorders' 40th Anniversary event
    Alexander also has a personal connection to the rare community.
  • Tickets and registration for the Rare Impact Awards and 40th Anniversary Celebration are available at rareimpact.org .
  • Additionally, the Usher Syndrome Society's Shine A Light on Usher  Syndrome photojournalism exhibit will be on display at the 2023 Rare Impact Awards.

Usher Syndrome Society hosting a free educational event to share the lived experience of going both deaf and blind from Usher syndrome (USH)

Retrieved on: 
Monday, December 5, 2022

BOSTON , Dec. 5, 2022 /PRNewswire/ -- The Usher Syndrome Society will host a free public event called This Is USH, an evening of conversation from 5:00 pm -7:00 pm at the Joseph B. Martin Conference Center at Harvard Medical School. This event is open to researchers, students, physicians, biotech and industry professionals, members of the USH community, and all interested persons to learn more about what it means to go both deaf and blind.

Key Points: 
  • BOSTON, Dec. 5, 2022 /PRNewswire/ -- The Usher Syndrome Society will host a free public event called This Is USH, an evening of conversation from 5:00 pm -7:00 pm at the Joseph B. Martin Conference Center at Harvard Medical School.
  • The Usher Syndrome Society is a non-profit organization that uses storytelling and educational events to raise awareness and funds for treatments and a cure for Usher syndrome.
  • Steven is on a mission to change the stigma that surrounds people who are blind and/or deaf and who also identifies with the LGBTQ+ and Black communities.
  • Rebecca Alexanderis an author, psychotherapist, disability rights advocate, and extreme athlete who is almost completely blind and deaf.

Simulated Dining Experience Raises Over $12,0000 to Benefit Those with Usher Syndrome

Retrieved on: 
Monday, November 21, 2022

HOLMDEL, N.J., Nov. 21, 2022 /PRNewswire/ -- On November 15th, 2022, Patricia's of Holmdel collaborated with Taylor Maxwell of Resources Real Estate to host "Light the Night," a simulated dining experience in honor of Ava Bullis's 17th birthday. The noteworthy event offered a simulated three course menu that allowed patrons an intimate opportunity to experience the world of DeafBlindness. Attendees were guided through the dishes, highlighting the aromas, ingredients, and pairings. The night was filled with delicious food, balloons from Weliketoparty, education, and celebration. With the support of all its customers, Patricia's of Holmdel & Taylor Maxwell managed to raise $12,240 which will go towards the gift of summer camp, through Ava's nonprofit, Ava's Voice. 

Key Points: 
  • The noteworthy event offered a simulated three course menu that allowed patrons an intimate opportunity to experience the world of DeafBlindness.
  • "We are honored to host this event for Ava and all those in the Usher Syndrome community."
  • The mission of Ava's Voice is to empower youth with Usher syndrome and to educate families and school communities.
  • The USHthis Camp is a youth summer camp operated by Ava's Voice to connect families with Usher syndrome and to advocate for access.

Foundation Launching its Largest Natural History Study to Date for 1,500 People with Inherited Retinal Diseases Caused by Rare Mutated Genes

Retrieved on: 
Thursday, November 10, 2022

COLUMBIA, Md., Nov. 10, 2022 /PRNewswire/ -- The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for blinding retinal diseases, is committing at least $8.6 million for its Uni-Rare Study, a new natural history study for approximately 1,500 people with one of more than 300 rare genes associated with inherited retinal diseases (IRDs) including: retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions.

Key Points: 
  • "A majority of the 300 genes linked to inherited retinal diseases have not been well characterized in the clinic.
  • "We are excited to launch a highly inclusive study to benefit a large segment of the population affected by IRDs, which are so genetically diverse."
  • Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

RD Fund Participates in a €75 Million Series B for SparingVision

Retrieved on: 
Thursday, September 15, 2022

RALEIGH, N.C., Sept. 15, 2022 /PRNewswire/ -- Today, the RD Fund (Retinal Degeneration Fund) – the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments, and cures for the entire spectrum of retinal degenerative diseases – announces its participation in a €75 million Series B financing for SparingVision. The round was co-led by Jeito Capital and UPMC Enterprises, with participation from 4BIO Capital, Bpifrance, Ysios Capital, and the RD Fund.

Key Points: 
  • RALEIGH, N.C., Sept. 15, 2022 /PRNewswire/ -- Today, the RD Fund (Retinal Degeneration Fund) the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments, and cures for the entire spectrum of retinal degenerative diseases announces its participation in a 75 million Series B financing for SparingVision .
  • The round was co-led by Jeito Capital and UPMC Enterprises , with participation from 4BIO Capital, Bpifrance , Ysios Capital, and the RD Fund.
  • SparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments.
  • "Our investment reflects the long-term funding and investment strategies of the Foundation and the RD Fund to bring forward a pipeline of novel therapeutics, including SparingVision's genetic medicines," said Rusty Kelley, PhD, MBA, managing director, RD Fund and SparingVision board director.

SparingVision Raises €75 Million Series B to Continue Building World-Leading Portfolio of Genomic Medicines for Ocular Diseases

Retrieved on: 
Wednesday, September 14, 2022

SparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments.

Key Points: 
  • SparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments.
  • Leveraging its unparalleled understanding of retinal diseases, SparingVision has built the worlds most compelling portfolio of synergistic cutting-edge gene therapy and genome editing treatments for inherited retinal diseases (IRDs).
  • The Company also has a strategic collaboration with Intellia Therapeutics (NASDAQ: NTLA) to develop novel genome editing-based treatments for ocular diseases utilizing CRISPR-Cas9 technology.
  • Inherited retinal diseases are a group of progressive eye conditions that can cause severe vision loss and, in certain cases, lead to total blindness.