Research & Updates
AI-curated articles covering the latest developments in inherited retinal disease research, clinical trials, treatments, and news.
Showing 9 of 425 articles
A New Hope for Bestrophinopathies: Opus Genetics Launches OPGx-BEST1 Clinical Trial
Opus Genetics is initiating a Phase 1 clinical trial (NCT07185256) for OPGx-BEST1, a novel gene therapy aimed at treating Best Vitelliform Macular Dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB). This trial will evaluate the safety, tolerability, and preliminary efficacy of a subretinal injection of OPGx-BEST1 over five years, offering a potential new treatment pathway for these inherited retinal diseases.
Unlocking the Secrets of Retinal Diseases: A New Study Seeks Cell Samples to Advance Research
The National Eye Institute (NEI) is recruiting participants for a vital study, NCT01432847, aimed at collecting cell samples from individuals with retinal diseases like Best disease, Late-Onset Retinal Degeneration (L-ORD), and Age-Related Macular Degeneration (AMD), as well as healthy volunteers. This research is crucial for understanding these conditions at a cellular level, paving the way for future diagnostic tools and treatments.
Unlocking Insights: A Decade-Long Study to Understand Congenital Muscle Diseases
ClearSight is excited to share details about a crucial, ongoing study, NCT01403402, focused on gathering vital patient and family-reported medical information for a wide range of congenital muscle diseases. This long-term research aims to improve understanding of these complex conditions and ultimately enhance patient care.
PYC Therapeutics Launches First-in-Human Trial for OPA1-Associated Autosomal Dominant Optic Atrophy (ADOA)
PYC Therapeutics has initiated a Phase 1 clinical trial (NCT06461286) to evaluate a new investigational treatment, PYC-001, for Autosomal Dominant Optic Atrophy (ADOA) caused by OPA1 gene mutations. This 'first-in-human' study aims to assess the safety and tolerability of PYC-001, offering new hope for individuals living with this progressive vision loss condition.
A New Hope for Autosomal Dominant Optic Atrophy: PYC-001 Clinical Trial Begins Recruitment
ClearSight is excited to announce the launch of a new Phase 1 clinical trial for PYC-001, a potential new treatment for Autosomal Dominant Optic Atrophy (ADOA) caused by OPA1 gene mutations. This trial, sponsored by PYC Therapeutics, will assess the safety and optimal dosing of PYC-001 eye injections, offering a glimmer of hope for individuals living with this progressive vision loss condition.
New Clinical Trial Explores Vitamin B3 as Potential Treatment for Dominant Optic Atrophy (DOA)
A new Phase 2 clinical trial, sponsored by the University Hospital in Angers, France, is investigating the tolerance and efficacy of nicotinamide (vitamin B3) as a potential treatment for Dominant Optic Atrophy (DOA). This study, building on promising metabolomic research, offers a ray of hope for individuals living with this progressive vision-impairing condition for which no proven treatment currently exists.
Unlocking the Secrets of Mitochondrial Disorders: The NAMDC Registry and Biorepository
ClearSight reports on the North American Mitochondrial Disease Consortium (NAMDC) registry and biorepository, a vital initiative collecting patient data and biological samples to advance research into mitochondrial disorders and accelerate the development of new treatments.
A New Horizon for LHON Patients: Gene Therapy Trial Investigates NR082 for ND4 Mutations
ClearSight reports on a Phase 1 clinical trial (NCT05293626) by Neurophth Therapeutics Inc., exploring the safety and efficacy of NR082, a gene therapy, for Leber's Hereditary Optic Neuropathy (LHON) caused by ND4 mutations. This trial offers a potential new treatment avenue for a debilitating inherited eye condition.
Unlocking Early Detection: New 'MétabOCT' Study Explores Metabolic Clues in Leber Hereditary Optic Neuropathy
ClearSight is excited to share news about a pioneering clinical trial, 'MétabOCT' (NCT06682819), which aims to identify early biomarkers for Leber Hereditary Optic Neuropathy (LHON). This study, led by Hôpital Necker-Enfants Malades, is investigating whether specific metabolic profiles and vitamin levels can indicate optic nerve damage in individuals with LHON-associated genetic mutations even before vision loss occurs. This research holds significant promise for earlier diagnosis and interventi