Rare Diseases

CheckOrphan is the leading #news and information platform dedicated to #rarediseases and #orphandrugs

[Rare Diseases @CheckOrphan]

Neurocrine Biosciences Publishes Expert Perspectives on Comprehensive Assessment and Management of Hyperphagia in Prader-Willi Syndrome - For More Information Visit https://t.co/VKcX7Sdq28 @neurocrine #Prader_Willi_Syndrome #Rare_Diseases #Orphan_Drugs #Hyperphagia

[Rare Diseases @CheckOrphan]

Korro Reports Best-in-Class Preclinical Data for KRRO-111 showing Potential Benefits for Lung and Liver Symptoms of Alpha-1 Antitrypsin Deficiency - For More Information Visit https://t.co/UVW6zej0qx @KorroBio #AATD #Alpha_1_Antitrypsin_Deficiency #Rare_Diseases

[Rare Diseases @CheckOrphan]

BioNTech and Gilead Team Up to Advance Promising Pumitamig and Trodelvy Combination in Phase 3 Trial for Triple-Negative Breast Cancer - For More Information Visit https://t.co/BLcUZNFNUr @BioNTech_Group #TNBC @GileadSciences #Breast_Cancer #Rare_Diseases

[Rare Diseases @CheckOrphan]

Prilenia and Ferrer Announce Authorization for EU Initiation of Confirmatory PRECISE-HD Study of Pridopidine in Huntington's Disease - For More Information Visit https://t.co/YFjxKR6evX @PrileniaTx @ferrerforgood #Huntington_Disease #Rare_Diseases #Orphan_Drugs

[Rare Diseases @CheckOrphan]

Efgartigimod Improves Mobility and Daily Function in Young Woman With Refractory LEMS After Multiple Prior Treatments - For More Information Visit https://t.co/lob6OZxv7a @argenxglobal #LEMS #Rare_Diseases #Orphan_Drugs #Lambert_Eaton_Myasthenic_Syndrome

[Rare Diseases @CheckOrphan]

Savara Reports Molgramostim Improvements in Gas Transfer, Respiratory Quality of Life, and Surfactant Burden in aPAP Patients - For More Information Visit https://t.co/WAEOhmdDTr @SavaraPharma #aPAP #Autoimmune_Pulmonary_Alveolar_Proteinosis #Rare_Diseases

[Rare Diseases @CheckOrphan]

Opus Genetics Receives FDA Clearance for OPGx-RDH12 Gene Therapy to Treat Rare RDH12-Associated Leber Congenital Amaurosis - For More Information Visit https://t.co/Rp0fA1jTOk #Opus_Genetics #RDH12_LCA #FDA #Leber_Congenital_Amaurosis #Rare_Diseases #Orphan_Drugs

[Rare Diseases @CheckOrphan]

European Commission Approves KEYTRUDA® Plus Padcev® as Perioperative Treatment for Adults With Resectable Muscle-Invasive Bladder Cancer - For More Information Visit https://t.co/s9ubNKSt73 @Merck #MIBC #Bladder_Cancer #KEYTRUDA #Rare_Diseases #Orphan_Drugs

[Rare Diseases @CheckOrphan]

Dartmouth Study Links Indoor Fine-Particle Exposure to Changes in Tissue-Repair Signaling in Adults With Cystic Fibrosis - For More Information Visit https://t.co/qCkZ6dQjr3 @DHMCandClinics #Cystic_Fibrosis #CF #Dartmouth #Rare_Diseases #Orphan_Drugs #Lung_Diseases

[Rare Diseases @CheckOrphan]

Neurogene Announces Upcoming Presentations Highlighting Key Differentiators of NGN-401 for Rett Syndrome at the ESGCT Congress - For More Information Visit https://t.co/aYDmDDlUGY @NeurogeneInc #ESGCT #Rett_Syndrome #Rare_Diseases #Orphan_Drugs #Brain_Disorders

[Rare Diseases @CheckOrphan]

New Research from One Biosciences Shows Single-Cell Profiling Can Identify Which Ovarian Cancer Tumors are Predisposed to Chemotherapy Resistance - For More Information Visit https://t.co/4ED33ETXyf #Cancer #One_Biosciences #Ovarian_Cancer #Rare_Diseases #Orphan_Drugs

[Rare Diseases @CheckOrphan]

Medicovestor Announces FDA Fast Track Designation for MC001, Its First-in-Class Chemoimmunotherapy ADC for Pancreatic Cancer - For More Information Visit https://t.co/7oPVAIsIJL #Medicovestor #Rare_Diseases #FDA #Orphan_Drugs #Pancreatic_Cancer

[Rare Diseases @CheckOrphan]

Quoin Pharmaceuticals Announces Publication of Peer-Reviewed Paper on QRX003 for Netherton Syndrome in Dermatology and Therapy - For More Information Visit https://t.co/0lfv69H0QX @QuoinPharma #Orphan_Drugs #Netherton_Syndrome #Rare_Diseases #Skin_Disorders

[Rare Diseases @CheckOrphan]

Once-Weekly TransCon® CNP (Navepegritide) Receives Positive CHMP Opinion for the Treatment of Achondroplasia in Children - For More Information Visit https://t.co/3Jn57TXRvY @AscendisPharma #CHMP #EMA #Rare_Diseases #Achondroplasia #Orphan_Drugs

[Rare Diseases @CheckOrphan]

Propanc Biopharma Reports Promising Preclinical Findings for PRP in Overcoming Chemotherapy Resistance in Pancreatic Cancer - For More Information Visit https://t.co/RUvbxiQti1 @PropancBio #Rare_Diseases #Orphan_Drugs #Pancreatic_Cancer #Propanc #Cancer_Research

[Rare Diseases @CheckOrphan]

Sobi Receives Positive CHMP Opinion for TRYNGOLZA® (olezarsen) in the Treatment of Severe Hypertriglyceridemia (sHTG) - For More Information Visit https://t.co/DfutSWEMk8 #Sobi #sHTG #Rare_Diseases #CHMP #TRYNGOLZA #Severe_Hypertriglyceridemia #Orphan_Drugs

[Rare Diseases @CheckOrphan]

FDA Approves Roche’s Enspryng, a First-in-Class IL-6R Inhibitor, as the First and Only at-home Subcutaneous Treatment Option for Thyroid Eye Disease - For More Information Visit https://t.co/x7Q53gMYtq @Roche #Thyroid_Eye_Disease #Eye_Diseases #Rare_Diseases #Orphan_Drugs

[Rare Diseases @CheckOrphan]

Egetis Therapeutics Announces Publication of Largest Retrospective Study to Date on Tiratricol Treatment in Resistance to Thyroid Hormone Beta - For More Information Visit https://t.co/zJaPhF1cZA @Egetis_EGTX #RTH_beta #Resistance_to_Thyroid_Hormone_Beta

[Rare Diseases @CheckOrphan]

Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026 - For More Information Visit https://t.co/iV262pRjz0 #Opus_Genetics #Rare_Diseases #LCA5 #Orphan_Drugs #Leber_Congenital_Amaurosis #Eye_Disorders

[Rare Diseases @CheckOrphan]

Palvella Therapeutics to Present New Pediatric Analyses of QTORIN™ Rapamycin for Microcystic Lymphatic Malformations and cVMs at PeDRA Annual Conference - For More Information Visit https://t.co/Y2Kzmv50wf @PalvellaTX #Microcystic_Lymphatic_Malformations #cVMs