Канал: AMDA YouTube Channel

In Memoriam - Dr. Paul Plotz

In Memoriam - Dr. Paul Plotz

The History of the AMDA: 1995 - 2024

The History of the AMDA: 1995 - 2024

Pull For Pompe 2024 Photo Montage

Pull For Pompe 2024 Photo Montage

Astellas Gene Therapies Webinar: Evaluating gene therapies for Pompe disease

Astellas Gene Therapies Webinar: Evaluating gene therapies for Pompe disease

Development of Gene Therapy for Pompe Disease

Development of Gene Therapy for Pompe Disease

Spark Therapeutics Webinar- Trial Expectations

Spark Therapeutics Webinar- Trial Expectations

Defining Central Nervous System Abnormalities in Infantile and Late-Onset Pompe Disease Patients

Defining Central Nervous System Abnormalities in Infantile and Late-Onset Pompe Disease Patients

Lentiviral Gene Therapy for Pompe Disease

Lentiviral Gene Therapy for Pompe Disease

How To Manage LOPD Patients Diagnosed Through NBS

How To Manage LOPD Patients Diagnosed Through NBS

When A Hospital and A Patient Organization Work Together & The Value of Patient Reported Outcomes

When A Hospital and A Patient Organization Work Together & The Value of Patient Reported Outcomes

AMDA WEBINAR WITH SPARK THERAPEUTICS

AMDA WEBINAR WITH SPARK THERAPEUTICS

2001 AMDA POMPE CONFERENCE PART 3 OF 3

2001 AMDA POMPE CONFERENCE PART 3 OF 3

2001 AMDA POMPE CONFERENCE PART 2 OF 3

2001 AMDA POMPE CONFERENCE PART 2 OF 3

2001 AMDA POMPE CONFERENCE PART 1 OF 3

2001 AMDA POMPE CONFERENCE PART 1 OF 3

Insurance 101: Learning the Basics

Insurance 101: Learning the Basics

2015 AMDA POMPE CONFERENCE PART 8 OF 8

2015 AMDA POMPE CONFERENCE PART 8 OF 8

2015 AMDA POMPE CONFERENCE PART 7 OF 8

2015 AMDA POMPE CONFERENCE PART 7 OF 8

2015 AMDA POMPE CONFERENCE PART 6 OF 8

2015 AMDA POMPE CONFERENCE PART 6 OF 8

2015 AMDA POMPE CONFERENCE PART 5 OF 8

2015 AMDA POMPE CONFERENCE PART 5 OF 8

2015 AMDA POMPE CONFERENCE PART 4 OF 8

2015 AMDA POMPE CONFERENCE PART 4 OF 8

2015 AMDA POMPE CONFERENCE PART 3 OF 8

2015 AMDA POMPE CONFERENCE PART 3 OF 8

2015 AMDA POMPE CONFERENCE PART 2 OF 8

2015 AMDA POMPE CONFERENCE PART 2 OF 8

2015 AMDA POMPE CONFERENCE PART 1 OF 8

2015 AMDA POMPE CONFERENCE PART 1 OF 8

#13 EXERCISE AND NUTRITIONAL CONSIDERATIONS FOR THE TREATMENT OF POMPE DISEASE DR. MARK TARNOPOLSK.

#13 EXERCISE AND NUTRITIONAL CONSIDERATIONS FOR THE TREATMENT OF POMPE DISEASE DR. MARK TARNOPOLSK.

#14 RESPIRATORY MUSCLE TRAINING IN LATE ONSET POMPE DISEASE. DR. HARRISON JONES

#14 RESPIRATORY MUSCLE TRAINING IN LATE ONSET POMPE DISEASE. DR. HARRISON JONES

#15 NONINVASIVE RESPIRATORY MANAGEMENT OF POMPE DISEASE DR. JOHN BACH HD.

#15 NONINVASIVE RESPIRATORY MANAGEMENT OF POMPE DISEASE DR. JOHN BACH HD.

#16 MANAGEMENT AND CARE OF POMPE PATIENTS QUESTION AND ANSWER

#16 MANAGEMENT AND CARE OF POMPE PATIENTS QUESTION AND ANSWER

#17 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR ANS VAN DER PLOEG.

#17 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR ANS VAN DER PLOEG.

#18 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR. PASCAL LAFORÊT.

#18 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR. PASCAL LAFORÊT.

#19 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR. PRIYA KISHNANI.

#19 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR. PRIYA KISHNANI.

#20 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR. BENEDIKT SCHOSER.

#20 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE DR. BENEDIKT SCHOSER.

#21 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE OPEN DISCUSSION WITH AUDIENCE.

#21 EXPERT ROUND TABLE: 20 YEARS OF TREATMENT AND CARE OPEN DISCUSSION WITH AUDIENCE.

#22 THE IPA ERASMUS POMPE SURVEY: PAST, PRESENT, AND FUTURE DR. NADINE VAN DER BEEK.

#22 THE IPA ERASMUS POMPE SURVEY: PAST, PRESENT, AND FUTURE DR. NADINE VAN DER BEEK.

#25 NEXT GENERATION THERAPY: ENZYME REPLACEMENT THERAPY ERT DR NINA RABEN.

#25 NEXT GENERATION THERAPY: ENZYME REPLACEMENT THERAPY ERT DR NINA RABEN.

#26 HOW ENZYME REPLACEMENT THERAPY WORKS UPDATE ON AMICUS TRIALS WITH ERT ASSOCIATED WITH CHAPERONE

#26 HOW ENZYME REPLACEMENT THERAPY WORKS UPDATE ON AMICUS TRIALS WITH ERT ASSOCIATED WITH CHAPERONE

#27 UPDATE ON GENZYME'S NEO GAA DR. BENEDIKT SCHOSER.

#27 UPDATE ON GENZYME'S NEO GAA DR. BENEDIKT SCHOSER.

#28 PRECLINICAL STUDIES FOR ORAL ERT WITH A TOBACCO SEED DERIVED A RECOMBINANT ACID MAITASE DR FR

#28 PRECLINICAL STUDIES FOR ORAL ERT WITH A TOBACCO SEED DERIVED A RECOMBINANT ACID MAITASE DR FR

#29 SAFETY AND EFFICACY DATA USING VAL 1221 IN PATIENTS WITH LOPD DR. PRIYA KISHNANI.

#29 SAFETY AND EFFICACY DATA USING VAL 1221 IN PATIENTS WITH LOPD DR. PRIYA KISHNANI.

#30 NEXT GENERATION THERAPY: ENZYME REPLACEMENT THERAPY (ERT) QUESTIONS AND ANSWER.

#30 NEXT GENERATION THERAPY: ENZYME REPLACEMENT THERAPY (ERT) QUESTIONS AND ANSWER.

#31 NEXT GENERATION THERAPY: GENE THERAPY DR. ARNOLD REUSER.

#31 NEXT GENERATION THERAPY: GENE THERAPY DR. ARNOLD REUSER.

#32 BASICS OF GENE THERAPY DR. DWIGHT KOEBERL.

#32 BASICS OF GENE THERAPY DR. DWIGHT KOEBERL.

#33 AAV MEDIATED GENE THERAPY IN POMPE DISEASE: CLINICAL IMMUNOLOGY CONSIDERATIONS DR. BARRY BYRNE

#33 AAV MEDIATED GENE THERAPY IN POMPE DISEASE: CLINICAL IMMUNOLOGY CONSIDERATIONS DR. BARRY BYRNE

#34 LENTIVIRAL GENE THERAPY AND ANTISENSE OLIGONUCLEOTIDES FOR POMPE DISEASE DR PIM PIJNAPPEL.

#34 LENTIVIRAL GENE THERAPY AND ANTISENSE OLIGONUCLEOTIDES FOR POMPE DISEASE DR PIM PIJNAPPEL.

#35 GENE THERAPY FOR POMPE DISEASE: FROM BENCH TO BEDSIDE DR. GIUSEPPE RONZITTI.

#35 GENE THERAPY FOR POMPE DISEASE: FROM BENCH TO BEDSIDE DR. GIUSEPPE RONZITTI.

#36 NEXT GENERATION THERAPY: GENE THERAPY QUESTION AND ANSWER.

#36 NEXT GENERATION THERAPY: GENE THERAPY QUESTION AND ANSWER.

#1 CRASH COURSE IN POMPE DR. ARNOLD REUSER.

#1 CRASH COURSE IN POMPE DR. ARNOLD REUSER.

#2 NATURAL HISTORY VS NEW HISTORY WITH TREATMENT DR. PASCAL LAFORÊT.

#2 NATURAL HISTORY VS NEW HISTORY WITH TREATMENT DR. PASCAL LAFORÊT.

#3 NATURAL HISTORY VS NEW HISTORY: INFANTILE ONSET DR. PRIYA KISHNANI.

#3 NATURAL HISTORY VS NEW HISTORY: INFANTILE ONSET DR. PRIYA KISHNANI.

#4 NATURAL HISTORY VS NEW HISTORY: LATE ONSET DR. ANS VAN DER PLOEG

#4 NATURAL HISTORY VS NEW HISTORY: LATE ONSET DR. ANS VAN DER PLOEG

#5 NEWBORN SCREENING: TAIWAN EXPERIENCE DR. YIN HSIU CHIEN.

#5 NEWBORN SCREENING: TAIWAN EXPERIENCE DR. YIN HSIU CHIEN.

#6 CRASH COURSE IN POMPE QUESTION AND ANSWER.

#6 CRASH COURSE IN POMPE QUESTION AND ANSWER.

#7 INTEGRATIVE CARE OF POMPE PATIENTS QUESTION AND ANSWER DR. HANNERIEKE VAN DEN HOUT.

#7 INTEGRATIVE CARE OF POMPE PATIENTS QUESTION AND ANSWER DR. HANNERIEKE VAN DEN HOUT.

#8 CLINICAL TRIAL RELATED ISSUES FOR POMPE DISEASE DR. BENEDIKT SCHOSER.

#8 CLINICAL TRIAL RELATED ISSUES FOR POMPE DISEASE DR. BENEDIKT SCHOSER.

#9 PATIENT PERSPECTIVE ON CLINICAL TRIAL RYAN COLBURN.

#9 PATIENT PERSPECTIVE ON CLINICAL TRIAL RYAN COLBURN.

#10 PATIENT ORGANIZATION VIEW ON CLINICAL TRIALS RIA BROEKGAARDEN.

#10 PATIENT ORGANIZATION VIEW ON CLINICAL TRIALS RIA BROEKGAARDEN.

#11 THE BASICS OF CLINICAL TRIAL QUESTION AND ANSWER.

#11 THE BASICS OF CLINICAL TRIAL QUESTION AND ANSWER.

#12 IMPROVED MUSCLE FUNCTION IN A PHASE I, II CLINICAL TRIAL OF ALBUTEROL IN POMPE DISEASE DR. DWIG.

#12 IMPROVED MUSCLE FUNCTION IN A PHASE I, II CLINICAL TRIAL OF ALBUTEROL IN POMPE DISEASE DR. DWIG.

Aquatic Exercise Webinar

Aquatic Exercise Webinar

AMDA INTERNATIONAL POMPE DAY 2022

AMDA INTERNATIONAL POMPE DAY 2022

GENZYME'S NEXT GENERATION POMPE TREATMENT: CLINICAL DEVELOPMENT OVERVIEW

GENZYME'S NEXT GENERATION POMPE TREATMENT: CLINICAL DEVELOPMENT OVERVIEW

THE COMMON C.-32-13T G SPLICING VARIANT OF GAA GENE

THE COMMON C.-32-13T G SPLICING VARIANT OF GAA GENE

NATIONAL PATIENT-TEACHER REGISTERY: A NEW EDUCATIONAL CONCEPT

NATIONAL PATIENT-TEACHER REGISTERY: A NEW EDUCATIONAL CONCEPT

DR. PRIYA KISHNANI, AN INFORMATIVE SESSION ON THE COVID 19 VACCINE WITH QUESTIONS AND ANSWERS

DR. PRIYA KISHNANI, AN INFORMATIVE SESSION ON THE COVID 19 VACCINE WITH QUESTIONS AND ANSWERS

AUTOPHAGY AND IT'S ROLE IN POMPE DISEASE

AUTOPHAGY AND IT'S ROLE IN POMPE DISEASE

2011 AMDA POMPE CONFERENCE PART 1 OF 6

2011 AMDA POMPE CONFERENCE PART 1 OF 6

2011 AMDA POMPE CONFERENCE PART 2 OF 6

2011 AMDA POMPE CONFERENCE PART 2 OF 6

2011 AMDA POMPE CONFERENCE PART 6 OF 6

2011 AMDA POMPE CONFERENCE PART 6 OF 6

THE CONTRIBUTION OF THE IPA SURVEY TO OUR UNDERSTANDING OF THE NATURAL COURSE OF POMPE DISEASE

THE CONTRIBUTION OF THE IPA SURVEY TO OUR UNDERSTANDING OF THE NATURAL COURSE OF POMPE DISEASE

RESPIRATORY MUSCLE STRENGTH TRAINING IN POMPE DISEASE

RESPIRATORY MUSCLE STRENGTH TRAINING IN POMPE DISEASE

#24 IPA ERASMUS SURVEY AND POMPE REGISTRY QUESTION AND ANSWER

#24 IPA ERASMUS SURVEY AND POMPE REGISTRY QUESTION AND ANSWER

#39 STEM CELL AND MUSCLE REGENERATION IN POMPE DR. PIM PIJNAPPEL.

#39 STEM CELL AND MUSCLE REGENERATION IN POMPE DR. PIM PIJNAPPEL.

#40 FUTURE RESEARCH QUESTION AND ANSWER ENDING OF THE CONFERENCE

#40 FUTURE RESEARCH QUESTION AND ANSWER ENDING OF THE CONFERENCE

2011 AMDA POMPE CONFERENCE PART 5 OF 6

2011 AMDA POMPE CONFERENCE PART 5 OF 6

2006 AMDA POMPE CONFERENCE PART 2 OF 6

2006 AMDA POMPE CONFERENCE PART 2 OF 6

2006 AMDA POMPE CONFERENCE PART 1 OF 6

2006 AMDA POMPE CONFERENCE PART 1 OF 6

DESIGN AND PERFORMANCE OF CLINICAL TRIALS: AN OVERVIEW

DESIGN AND PERFORMANCE OF CLINICAL TRIALS: AN OVERVIEW

DIAPHRAGM DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS ACID ALPHA-GLUCOSIDASE

DIAPHRAGM DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS ACID ALPHA-GLUCOSIDASE

2006 AMDA POMPE CONFERENCE PART 5 OF 6

2006 AMDA POMPE CONFERENCE PART 5 OF 6

BMN-701: POTENTIALLY MORE EFFECTIVE TREATMENT OPTION FOR POMPE

BMN-701: POTENTIALLY MORE EFFECTIVE TREATMENT OPTION FOR POMPE

2006 AMDA POMPE CONFERENCE PART 4 OF 6

2006 AMDA POMPE CONFERENCE PART 4 OF 6

#37 SKELETAL MUSCLE ABNORMALITIES CAN BE REVERSED BY ENZYME REPLACEMENT THERAPY. DR. NINA RABE

#37 SKELETAL MUSCLE ABNORMALITIES CAN BE REVERSED BY ENZYME REPLACEMENT THERAPY. DR. NINA RABE

2011 AMDA POMPE CONFERENCE PART 3 OF 6

2011 AMDA POMPE CONFERENCE PART 3 OF 6

2011 AMDA POMPE CONFERENCE PART 4 OF 6

2011 AMDA POMPE CONFERENCE PART 4 OF 6

NMR IMAGING AND SPECTROSCOPIC TECHNIQUES FOR DIAGNOSIS, FOLLOW UP AND EVALUATION OF POMPE DISEASE

NMR IMAGING AND SPECTROSCOPIC TECHNIQUES FOR DIAGNOSIS, FOLLOW UP AND EVALUATION OF POMPE DISEASE

NEEDLE CORE MUSCLE BIOPSY

NEEDLE CORE MUSCLE BIOPSY

#38 TARGETING GLYCOGEN SYNTHASE GYS1 WITH ANTISENSE OLIGONUCLEOTIDE TREATMENT... DR.VIRGINIA KIMONIS

#38 TARGETING GLYCOGEN SYNTHASE GYS1 WITH ANTISENSE OLIGONUCLEOTIDE TREATMENT... DR.VIRGINIA KIMONIS

2006 AMDA POMPE CONFERENCE PART 6 OF 6

2006 AMDA POMPE CONFERENCE PART 6 OF 6

2006 AMDA POMPE CONFERENCE PART 3 OF 6

2006 AMDA POMPE CONFERENCE PART 3 OF 6

#23 REPORT FROM GENZYME'S POMPE REGISTRY DR. VIRGINIA KIMONIS

#23 REPORT FROM GENZYME'S POMPE REGISTRY DR. VIRGINIA KIMONIS

THANK YOU FROM THE AMDA/IPA INTERNATIONAL CONFERENCE 2019

THANK YOU FROM THE AMDA/IPA INTERNATIONAL CONFERENCE 2019

PCMA AND PULL FOR POMPE HISTORY

PCMA AND PULL FOR POMPE HISTORY

20 YEAR HISTORY OF THE AMDA, 1995-2015

20 YEAR HISTORY OF THE AMDA, 1995-2015