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Molecular Basis of Pulmonary Disease: Insights from Rare Lung Disorders - Respiratory Medicine (Paperback)
  • Molecular Basis of Pulmonary Disease: Insights from Rare Lung Disorders - Respiratory Medicine (Paperback)
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Molecular Basis of Pulmonary Disease: Insights from Rare Lung Disorders - Respiratory Medicine (Paperback)

(editor), (editor), (editor)
£199.99
Paperback 438 Pages / Published: 23/08/2016
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Dr. SharonRounds, theeditorforthisserieswhoinvitedustowriteabookonrare lungdiseases, developedtheideaafterattendingthe2004Lymphangioleiomyomatosis (LAM)Foundationannualresearchmeeting. Shewasakeynotespeakeratthatevent (duringhertenureasthepresidentoftheAmericanThoracicSociety)andwasw- nesstothepowerofpatientadvocacyandthemission-basedscienti ceffortthathad broughtthisrarediseaseofwomenfromobscuritytoclinicaltrialswithtargetedmol- ulartherapiesinunderadecade. Theprogressinpulmonaryalveolarproteinosis(PAP), pulmonaryalveolarmicrolithiasis(PAM), inheriteddisordersofsurfactantmetabolism, and pulmonary arterial hypertension, to name a few, has been no less astounding. Advanceshavecomefromthemostsurprisingdirections;fruit iesforLAM, gen- ically engineered mice made for other purposes for PAP, and groundbreaking hi- densitySNP(single-nucleotidepolymorphism)analysesdoneonahandfuloffamilies forPAM. Inmanycases, insightsintobiologygainedfromrarediseaseshaveinformed researchapproachesandtreatmentstrategiesformorecommondiseases;forexample, knowledgegainedfromthestudyofPAPabouttheroleofGM-CSFinthelunghas sparkedinterestintheuseofantiGM-CSFapproachestocontrolbothpulmonaryand extrapulmonaryin ammationinavarietyofdiseases. The ndingthatinterstitiallung diseasedevelopsinfamilieswithcytotoxicmutationsinsurfactantproteinC(SP-C), agenewhichisexpressedonlyinalveolartypecells, hasunderscoredtheimportance oftheintegrityofthealveolarepitheliuminthepathogenesisofparenchymal brosis. Opportunitiestoapproachlungdiseasepathogenesisfromthevantagepointofap- marymoleculardefectaregiftsfromnaturethatareuniquelyabundantamongtherare lungdisorders. WesalutetheNIHandtheNationalCenterforResearchResourcesfortheirvisionin facilitatingthetranslationofbasicresearchadvancesinrarelungdiseasesintoclinical realitythroughtheRareLungDiseaseConsortium, anetworkof13USandinter- tionalsitesthatiscurrentlyconductingclinicaltrialsandstudiesinLAM, alphaone antitrypsin de ciency, pediatric interstitial lung disease, and PAP. It has been a rare privilegetoworkonsuchfascinatingdiseaseswithsuchcapableinvestigatorsfromall overtheworldoverthepast6years. v vi Preface Theformatforthisvolumeisunique. Mostchaptershavebeenauthoredbyacli- cianandabasicscientistwhoareexpertinthediseasetopicandunderlyingmolecular defect, respectively. Theirchargewastofocusonthegeneticbasisandmolecularpat- genesisofdisease, animalmodels, clinicalfeatures, diagnosticapproach, conventional managementandtreatment, andfuturetherapeutictargetsanddirections. Theintentwas nottoprovideabroadoverview, butrathertoshedlightonthemolecularmechanisms thatevoketheclinicalpresentationandengendertreatmentstrategiesforeachdisease. Wehopethatthisapproachwillproveusefulforpulmonarycliniciansandscientists alike. Wethankourwives, Holly, Jean, andVicky, fortheirsupportandindulgencewith latenightemailsandwork- lledweekends, Dr. Roundsfortheinvitationtowritethe book, andalloftheauthorswhocontributed. FrancisMcCormack, MD RalphPanos, MD BruceTrapnell, MD Contents Preface. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . v Contributors. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . ix 1 AClinicalApproachtoRareLungDiseases. . . . . . . . . . . . . . . . 1 RalphJ. Panos 2 ClinicalTrialsforRareLungDiseases. . . . . . . . . . . . . . . . . . . 31 JeffreyKrischer 3 IdiopathicandFamilialPulmonaryArterialHypertension . . . . . . . . 39 JeanM. Elwing, GailH. Deutsch, WilliamC. Nichols, andTimothyD. LeCras 4 Lymphangioleiomyomatosis. . . . . . . . . . . . . .

Publisher: Humana Press Inc.
ISBN: 9781493960811
Number of pages: 438
Weight: 1157 g
Dimensions: 254 x 178 mm
Edition: Softcover reprint of the original 1st ed. 201


MEDIA REVIEWS

From the reviews:

"This unique book attempts to briefly review the clinical manifestations and evolution of a few uncommon, mostly very rare pulmonary disorders, some of which even subspecialist pulmonologists rarely or never encounter during their careers. ... The editors derive insight and input from contributors of eclectic knowledge and experience. ... Certainly, the audience would be limited to basic scientists and a modicum of clinicians with unique interests. ... The book achieves its likely intended goals well. ... a potentially useful addition to my medical library." (Joel C. Seidman, Doody's Review Service, July, 2010)

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