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Pharming To Develop Leniolisib For Additional PIDs Beyond APDS; Phase 2 Trial To Initiate In Q2

Dutch biopharmaceutical company Pharming Group N.V. (PHAR) announced Wednesday its plans to develop leniolisib for additional primary immunodeficiencies or PIDs beyond activated phosphoinositide 3-kinase delta syndrome or APDS.

Beth israel lahey healthMore such health newsClinical immunodeficiency programPharming groupDrug administrationPhase 2 trialPrimary immunodeficienciesHarming leniolisib

FDA OKs Iptacopan to Treat Paroxysmal Nocturnal Hemoglobinuria

This marks the first oral monotherapy approved for the rare blood disorder.

United statesVinod pullarkatDrug administrationCity of hope cancer centerHope cancer centerRisk evaluationMitigation strategyParoxysmal nocturnal hemoglobinuriaNh paroxysmal nocturnal hemoglobinuriaParoxysmal nocturnal haemoglobinuriaPrimary immunodeficiencyPrimary immunodeficienciesNt specialtyEad and neck surgery ent specialityCovid 19019 novel coronavirus

Telethon Foundation Will Make and Distribute ADA-SCID Drug

The initiative will ensure the availability of a drug that otherwise would have been abandoned as unprofitable.

Alessandro aiutiFrancesca pasinelliFoundation stepsNational health service for italianScientific institute for researchTelethon foundationSan raffaele telethon institute for gene therapyEuropean unionEuropean medicines agencyPediatric immunohematology unitScientific instituteSan raffaele hospitalSan raffaele telethon instituteGene therapyOrchard therapeuticsSan raffaele

No-Biopsy Approach to Celiac Disease Diagnosis Safe for Some

For adults with suspected celiac disease and no IgA deficiency, serology alone appears safe and reliable for diagnosis.

Centre for coeliac diseaseCarolina ciacciCoeliac diseaseGiovanni di dioLancet gastroenterologyCeliac diseaseCeliac sprueGluten sensitive enteropathyPrue celiacImmunoglobulina deficiencyElective immunoglobulina deficiency sigadGa deficiencyGa subclass deficiencyMmunoglobulina subclass deficiencyPrimary immunodeficiencyPrimary immunodeficiencies

Horizon Therapeutics plc Announces 2023 RAREis Global Advocate Grant Recipients

Horizon Therapeutics plc Announces 2023 RAREis Global Advocate Grant Recipients
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Nederlandse leverpatienten verenigingEimear rigbyAssociazione italiana sindromeDistrofia brasilMatt fleschRareis instagramMichelle rivasA italia famiglieBeck fahrner syndrome foundationRare disease ghana initiativeChordoma foundationSyndrome foundationInternational gaucher allianceErdheim chester disease global allianceInternational patient organisation for primary immunodeficienciesCholestasis network