Канал: Rare Disease Report

Jasmine Zain, MD, Highlights Current & Upcoming T-Cell Lymphomas Treatments

Jasmine Zain, MD, Highlights Current & Upcoming T-Cell Lymphomas Treatments

Michael Jordan, MD, Differentiates HLH Treatment in Pediatric Patients Versus Adults with Emapalumab

Michael Jordan, MD, Differentiates HLH Treatment in Pediatric Patients Versus Adults with Emapalumab

Germ Cell Tumors Have the Potential to Become Leukemia

Germ Cell Tumors Have the Potential to Become Leukemia

What Are Next Steps for Retrotope's RT001?

What Are Next Steps for Retrotope's RT001?

What Makes RT001 Unique?

What Makes RT001 Unique?

How FDA’s Breakthrough Therapy Designation Program Changed the Rare Disease Space

How FDA’s Breakthrough Therapy Designation Program Changed the Rare Disease Space

Rare Tumor Patient Engagement Network: Getting Patients Involved in Cancer Research

Rare Tumor Patient Engagement Network: Getting Patients Involved in Cancer Research

Fibrolamellar Cancer Foundation President on What’s Next for Rare Cancers

Fibrolamellar Cancer Foundation President on What’s Next for Rare Cancers

Addressing Diagnosis Challenges in the Rare Disease Space

Addressing Diagnosis Challenges in the Rare Disease Space

AI: The Future for Rare Disease Treatments

AI: The Future for Rare Disease Treatments

Humility- "The Best Characteristic in Medicine"

Humility- "The Best Characteristic in Medicine"

Using Genomic Technology to Stratify Pediatric Cancer Patients

Using Genomic Technology to Stratify Pediatric Cancer Patients

FDA Approves First Chemotherapy-Free Treatment for Waldenstrom's Macroglobulinemia

FDA Approves First Chemotherapy-Free Treatment for Waldenstrom's Macroglobulinemia

Understanding Genetics to Develop Future Treatments for Rare Cancers

Understanding Genetics to Develop Future Treatments for Rare Cancers

Pediatric MDS Update with Dr. Inga Hofmann Zhang

Pediatric MDS Update with Dr. Inga Hofmann Zhang

MDS/AML Overlap and Novel Therapies with Dr. Naval Daver

MDS/AML Overlap and Novel Therapies with Dr. Naval Daver

PNH Treatment Update

PNH Treatment Update

MDS/MPN Overlap Syndromes and Myelofibrosis

MDS/MPN Overlap Syndromes and Myelofibrosis

Treating Children with Aplastic Anemia

Treating Children with Aplastic Anemia

Challenges for Treating Patients with Rare Cancers

Challenges for Treating Patients with Rare Cancers

Molecular Events—Not Time—Define Pediatric Oncology Outcomes

Molecular Events—Not Time—Define Pediatric Oncology Outcomes

Disparities Between Pediatric and Adult Cancers

Disparities Between Pediatric and Adult Cancers

Uncovering Rare Genetic Factors with Multiplex Germline Testing

Uncovering Rare Genetic Factors with Multiplex Germline Testing

Back to the Drawing Board: The Pivotal Role of Clinical Trials in Lymphoma Treatments

Back to the Drawing Board: The Pivotal Role of Clinical Trials in Lymphoma Treatments

A Family Affair: Multiplex Germline Testing in Melanoma

A Family Affair: Multiplex Germline Testing in Melanoma

Advantages of Brentuximab Vedotin and Chemotherapy Combination for Advanced Stage Hodgkin Lymphoma

Advantages of Brentuximab Vedotin and Chemotherapy Combination for Advanced Stage Hodgkin Lymphoma

Efficacy of Ibrutinib and Rituximab Combination for Waldenström's Macroglobulinemia

Efficacy of Ibrutinib and Rituximab Combination for Waldenström's Macroglobulinemia

Benefits of CART-T Therapies to Treat Rare Cancers

Benefits of CART-T Therapies to Treat Rare Cancers

Providing Pediatric Cancer Patients With Tools to Boost Resiliency During Treatment

Providing Pediatric Cancer Patients With Tools to Boost Resiliency During Treatment

Brain Tumors and Team Approaches

Brain Tumors and Team Approaches

Experience: The Most Valuable Resource in Rare Diseases

Experience: The Most Valuable Resource in Rare Diseases

Team Approaches with Brain Tumors and Other Rare Cancers

Team Approaches with Brain Tumors and Other Rare Cancers

Darcy Tannehill: AL Amyloidosis Diagnosis

Darcy Tannehill: AL Amyloidosis Diagnosis

Darcy Tannehill: Early Presentation of AL Amyloidosis

Darcy Tannehill: Early Presentation of AL Amyloidosis

Darcy Tannehill: Treatment Gaps in AL Amyloidosis

Darcy Tannehill: Treatment Gaps in AL Amyloidosis

Darcy Tannehill: Living with a Rare Disease

Darcy Tannehill: Living with a Rare Disease

FoundationOne CDx: A Broad Companion Diagnostic Test for Cancer Tumors

FoundationOne CDx: A Broad Companion Diagnostic Test for Cancer Tumors

How GSSI is Fighting Gastroparesis

How GSSI is Fighting Gastroparesis

A Caregiver's Perspective: Light Chain (AL) Amyloidosis Treatment

A Caregiver's Perspective: Light Chain (AL) Amyloidosis Treatment

The Effect of AL Amyloidosis

The Effect of AL Amyloidosis

FCS Diet and Dangers

FCS Diet and Dangers

Managing hATTR Amyloidosis

Managing hATTR Amyloidosis

Cofactors and Concerns in FCS Treatments

Cofactors and Concerns in FCS Treatments

Cofactors and Concerns in FCS Treatment

Cofactors and Concerns in FCS Treatment

Hypertriglyceridemia in Connection to FCS

Hypertriglyceridemia in Connection to FCS

Emerging FCS Treatments and Research

Emerging FCS Treatments and Research

The Importance of Rare Disease Awareness Among Physicians

The Importance of Rare Disease Awareness Among Physicians

FCS and the Need for Treatments

FCS and the Need for Treatments

The Importance of Proper FCS Nutrition

The Importance of Proper FCS Nutrition

Understanding the Genetics of hATTR Amyloidosis

Understanding the Genetics of hATTR Amyloidosis

Rare Disease Report

Rare Disease Report

New Resources Available to IPF Patients

New Resources Available to IPF Patients

2018 ALA LUNG FORCE Expos

2018 ALA LUNG FORCE Expos

IPF KOL Discusses Current and Emerging Therapies

IPF KOL Discusses Current and Emerging Therapies

Mestinon and Other Myasthenia Gravis Therapies

Mestinon and Other Myasthenia Gravis Therapies

How Raising Awareness is Changing the Myasthenia Gravis Landscape

How Raising Awareness is Changing the Myasthenia Gravis Landscape

Myasthenia Gravis: The Road to Diagnosis

Myasthenia Gravis: The Road to Diagnosis

Symptoms and Disease Course of Myasthenia Gravis

Symptoms and Disease Course of Myasthenia Gravis

Researching MPS Therapies with Dr. Patricia Dickson of LA BioMed

Researching MPS Therapies with Dr. Patricia Dickson of LA BioMed

The Importance of Researcher Involvement in the Rare Disease Community

The Importance of Researcher Involvement in the Rare Disease Community

Rare Disease Report Sits Down with Hartmann Wellhoefer, M.D. of Shire

Rare Disease Report Sits Down with Hartmann Wellhoefer, M.D. of Shire

Feeling a Sense of Urgency in Sanfilippo Therapy Development

Feeling a Sense of Urgency in Sanfilippo Therapy Development

Is There A Link Between Gaucher and Parkinsons?

Is There A Link Between Gaucher and Parkinsons?

John Crowley of Amicus Discusses Newly-Released Positive Data

John Crowley of Amicus Discusses Newly-Released Positive Data

RDR Takes the #ALSPepperChallenge

RDR Takes the #ALSPepperChallenge

Rare Connections: Clinical Trials in Cholangiocarcinoma

Rare Connections: Clinical Trials in Cholangiocarcinoma

Rare Connections: Cholangiocarcinoma Patients Finding Support

Rare Connections: Cholangiocarcinoma Patients Finding Support

Rare Connections: Building a Cholangiocarcinoma Care Team

Rare Connections: Building a Cholangiocarcinoma Care Team

Rare Connections: Unmet Needs in Cholangiocarcinoma

Rare Connections: Unmet Needs in Cholangiocarcinoma

Rare Connections: Diagnosing Cholangiocarcinoma Early

Rare Connections: Diagnosing Cholangiocarcinoma Early

Explaining the Challenges of Pediatric Ulcerative Colitis

Explaining the Challenges of Pediatric Ulcerative Colitis

Should Adcetris be Evaluated as a Combination Therapy?

Should Adcetris be Evaluated as a Combination Therapy?

Emmaus Presents Endari Data at ASH

Emmaus Presents Endari Data at ASH

Seattle Genetics CEO on Front-Line Therapy for Advanced Hodgkin Lymphoma

Seattle Genetics CEO on Front-Line Therapy for Advanced Hodgkin Lymphoma

Guy Young, MD, on the Recent Revolutions in Hemophilia Treatment

Guy Young, MD, on the Recent Revolutions in Hemophilia Treatment

MPN Expert Ruben Mesa on the Anemia-Based Data at ASH

MPN Expert Ruben Mesa on the Anemia-Based Data at ASH

Rigel CEO Encouraged by Company's Progress and FDA Activity

Rigel CEO Encouraged by Company's Progress and FDA Activity

CMO of Catalyst Biosciences Explains the Current State of Hemophilia

CMO of Catalyst Biosciences Explains the Current State of Hemophilia

Oncology Expert Explains Why These Are Exciting Times in Rare Cancers

Oncology Expert Explains Why These Are Exciting Times in Rare Cancers

Maha Radhakrishnan, M.D., Explains the Unmet Need in Orphan Diseases Like CAgD

Maha Radhakrishnan, M.D., Explains the Unmet Need in Orphan Diseases Like CAgD

UW General Surgery Resident Explains T Cell Immunotherapy in Fibrolamellar Carcinoma

UW General Surgery Resident Explains T Cell Immunotherapy in Fibrolamellar Carcinoma

Can Neratinib Be Effective in Liver Cancer

Can Neratinib Be Effective in Liver Cancer

Using a Hyperbaric Chamber for Duchenne Muscular Dystrophy

Using a Hyperbaric Chamber for Duchenne Muscular Dystrophy

Praveen Sethupathy, PhD Explores the Fibrolamellar Space

Praveen Sethupathy, PhD Explores the Fibrolamellar Space

Why is the #MyCauseMyCleats So Important? Jim Raffone Explains

Why is the #MyCauseMyCleats So Important? Jim Raffone Explains

The Sickle Cell Association of NJ Discusses Awareness and Delayed Diagnosis

The Sickle Cell Association of NJ Discusses Awareness and Delayed Diagnosis

Richard Bryce of Puma Biotechnology Talks Neratinib in Fibrolamellar

Richard Bryce of Puma Biotechnology Talks Neratinib in Fibrolamellar

Len Walt of SOBI Talks Still's Disease and Anakinra

Len Walt of SOBI Talks Still's Disease and Anakinra

Jim Kelly Talks About the Death of His Son

Jim Kelly Talks About the Death of His Son

John Gordan of USCF Discusses The Data Presented at the FCF Summit

John Gordan of USCF Discusses The Data Presented at the FCF Summit

Fibrolamellar Isn't Rare to John Gordan of UCSF

Fibrolamellar Isn't Rare to John Gordan of UCSF

What Does the Future Hold for Fibrolamellar Cancer?

What Does the Future Hold for Fibrolamellar Cancer?

Ghassan Abou-Alfa Discusses Fibrolamellar Cancer

Ghassan Abou-Alfa Discusses Fibrolamellar Cancer

Difficulties in Working with Small Patient Populations with John Scott of UW

Difficulties in Working with Small Patient Populations with John Scott of UW

Why is Immunotherapy So Empowering for Cancer Patients? With Jill O'Donnell-Tormey

Why is Immunotherapy So Empowering for Cancer Patients? With Jill O'Donnell-Tormey

Jill O'Donnell-Tormey Explains Immunology in Rare Cancers

Jill O'Donnell-Tormey Explains Immunology in Rare Cancers

Rare Action Network - Connecticut

Rare Action Network - Connecticut

Diagnosing Cluster Headaches

Diagnosing Cluster Headaches

Cluster Headache Support Group

Cluster Headache Support Group

What are Cluster Headaches

What are Cluster Headaches

Len Walt of Sobi Explains NOMID

Len Walt of Sobi Explains NOMID

Advocacy 101: Making Goals for your Scientific / Medical Advisory Board

Advocacy 101: Making Goals for your Scientific / Medical Advisory Board

Aplastic Anemia & MDS International Foundation

Aplastic Anemia & MDS International Foundation

Emergency Packs for Rare Disease Patients

Emergency Packs for Rare Disease Patients

President of Sobi in North America Discusses Company's Role in Rare

President of Sobi in North America Discusses Company's Role in Rare

Marjorie Guthrie's Impact on Rare Disease Advocacy

Marjorie Guthrie's Impact on Rare Disease Advocacy

Patient Engagement at the FDA

Patient Engagement at the FDA

Hearing Aids and Parental Concerns

Hearing Aids and Parental Concerns

Chemotherapy, Hearing Loss, and School Bureaucracy

Chemotherapy, Hearing Loss, and School Bureaucracy

The Origins of Sodium Thiosulfate as an Otoprotective Agent

The Origins of Sodium Thiosulfate as an Otoprotective Agent

Orphan Drugs, Exclusivity, and Suing the FDA

Orphan Drugs, Exclusivity, and Suing the FDA

The Origins of Sodium Thiosulfate as an Otoprotective Agent - Extended Interview

The Origins of Sodium Thiosulfate as an Otoprotective Agent - Extended Interview

Hearing Aids and Parental Concerns

Hearing Aids and Parental Concerns

Hearing Aids and Strong-willed 5-Year Olds

Hearing Aids and Strong-willed 5-Year Olds

The Real Cost of Hearing Aids

The Real Cost of Hearing Aids

Mother and Son with Cisplatin-induced Hearing Loss

Mother and Son with Cisplatin-induced Hearing Loss

Hearing Loss is Exhausting

Hearing Loss is Exhausting

Cancer Treatment and Hearing Loss: Winning the Battle But Losing The War

Cancer Treatment and Hearing Loss: Winning the Battle But Losing The War

8 Friends Needed to Help 1 Hearing-impaired Person

8 Friends Needed to Help 1 Hearing-impaired Person

Hearing Loss, Isolation, and Ludwig van Beethoven

Hearing Loss, Isolation, and Ludwig van Beethoven

Louise Vetter Stresses the Importance of Storytelling

Louise Vetter Stresses the Importance of Storytelling

Louise Vetter Previews "Her Mother's Daughter"

Louise Vetter Previews "Her Mother's Daughter"

A Little Hearing Loss is a Big Deal

A Little Hearing Loss is a Big Deal

The Trouble with Recognizing Milestones in the Rare Disease Community

The Trouble with Recognizing Milestones in the Rare Disease Community

Observing 50 Years with the HDSA

Observing 50 Years with the HDSA

Are Neurological Diseases Life-Threatening?

Are Neurological Diseases Life-Threatening?

Quality of Life in Children with Hearing Loss Following Chemotherapy

Quality of Life in Children with Hearing Loss Following Chemotherapy

Cisplatin-associated Hearing Loss in Children - The Past, Present, and Future

Cisplatin-associated Hearing Loss in Children - The Past, Present, and Future

Otoprotectant Properties of Sodium Thiosulfate: Results of the SIOPEL 6 Study

Otoprotectant Properties of Sodium Thiosulfate: Results of the SIOPEL 6 Study

Arginase-1 Deficiency and the French Fries Diet

Arginase-1 Deficiency and the French Fries Diet

Differences in Leukodystrophy Presentations

Differences in Leukodystrophy Presentations

Pharma, the FDA, and the Patient's Voice

Pharma, the FDA, and the Patient's Voice

Leukodystrophy Expert Discusses H-ABC

Leukodystrophy Expert Discusses H-ABC

An App to Help Advocates Groups Collect Patient Data

An App to Help Advocates Groups Collect Patient Data

Make-A-Wish NJ CEO Gets Help From Disney Imagineers

Make-A-Wish NJ CEO Gets Help From Disney Imagineers

Take a Tour of Make-A-Wish NJ's Wishing Place

Take a Tour of Make-A-Wish NJ's Wishing Place

monARC - An App for Clinical Trial Patients

monARC - An App for Clinical Trial Patients

Whole Exome Sequencing for Rare Diseases

Whole Exome Sequencing for Rare Diseases

Training Seizure Dogs

Training Seizure Dogs

Genetic Data Sharing

Genetic Data Sharing

ALS Expert Mary Sedarous Stresses the Importance of Time (2 of 2)

ALS Expert Mary Sedarous Stresses the Importance of Time (2 of 2)

ALS Expert Mary Sedarous Stresses the Importance of Time (1 of 2)

ALS Expert Mary Sedarous Stresses the Importance of Time (1 of 2)

What Clinicians Need to Know About Lipodystrophy

What Clinicians Need to Know About Lipodystrophy

MERRF Syndrome Mom Discusses Why Adopting Her Daughter Led to a Faster Diagnosis

MERRF Syndrome Mom Discusses Why Adopting Her Daughter Led to a Faster Diagnosis

MERRF Syndrome Mom Gets Help from the Internet

MERRF Syndrome Mom Gets Help from the Internet

ALS Expert Mary Sedarous Discusses Symptom Management

ALS Expert Mary Sedarous Discusses Symptom Management

RARE Champion of Hope 2017 - Collaboration in Advocacy

RARE Champion of Hope 2017 - Collaboration in Advocacy

Epilepsy Awareness Day at Disneyland

Epilepsy Awareness Day at Disneyland

ALS Expert Mary Sedarous is Hopeful for FDA-Approved Radicava

ALS Expert Mary Sedarous is Hopeful for FDA-Approved Radicava

Team Impact - Bringing Athletes and Patients Together

Team Impact - Bringing Athletes and Patients Together

JAR of Hope Raises Duchenne Awareness from the Grand Canyon

JAR of Hope Raises Duchenne Awareness from the Grand Canyon

Dup15q Mom Stresses the Importance of the Internet

Dup15q Mom Stresses the Importance of the Internet

Tribute to Champions of Hope 2017 - The Blue Carpet

Tribute to Champions of Hope 2017 - The Blue Carpet

Executive Director of TargetCancer Talks Cholangiocarcinoma

Executive Director of TargetCancer Talks Cholangiocarcinoma

Meet the Creators of Disorder: The Rare Disease Film Festival

Meet the Creators of Disorder: The Rare Disease Film Festival

A Paradigm Shift in Clinical Trial is Occurring

A Paradigm Shift in Clinical Trial is Occurring

Global Genes Tribute - Termeer

Global Genes Tribute - Termeer

Global Genes Tribute - Sparsh Shah

Global Genes Tribute - Sparsh Shah

Global Genes Tribute - National Anthem

Global Genes Tribute - National Anthem

Global Genes Tribute - We Carry Kevin

Global Genes Tribute - We Carry Kevin

Some1 Like You

Some1 Like You

Rare Disease Champions of Hope

Rare Disease Champions of Hope

Kind-Doll - Introduction

Kind-Doll - Introduction

Photography Book Focused on Rare Diseases

Photography Book Focused on Rare Diseases

Doll Kind: Empowering Children to Spread Kindness

Doll Kind: Empowering Children to Spread Kindness

hayleybrooks-45sec

hayleybrooks-45sec

How to Participate in the Rare Genomes Project

How to Participate in the Rare Genomes Project

George Yohrling, PhD Discusses Treatment Options for HD

George Yohrling, PhD Discusses Treatment Options for HD

Long Island Mother and Mast Cell Patient Fights for School Bus Access

Long Island Mother and Mast Cell Patient Fights for School Bus Access

HD Experts Talk About Being Patient-Facing

HD Experts Talk About Being Patient-Facing

Daughter of an ALS Patient Shares Her Experiences

Daughter of an ALS Patient Shares Her Experiences

Hall of Fame Quarterback Jim Kelly Stresses the Need for Early Detection

Hall of Fame Quarterback Jim Kelly Stresses the Need for Early Detection

Hall of Fame Quarterback Jim Kelly Discusses Krabbe Advocacy

Hall of Fame Quarterback Jim Kelly Discusses Krabbe Advocacy

Social Worker Anne Cooney Describes the Journey of an ALS Patient

Social Worker Anne Cooney Describes the Journey of an ALS Patient

ALS Expert Mary Sedarous, M.D. Discusses Early Presentation

ALS Expert Mary Sedarous, M.D. Discusses Early Presentation

The Importance of the HDSA

The Importance of the HDSA

Exploring HD - Social

Exploring HD - Social

Exploring Huntington’s Disease with the HDSA

Exploring Huntington’s Disease with the HDSA

Gail Houseman Explains ALS Pathophysiology and Progression

Gail Houseman Explains ALS Pathophysiology and Progression

PLS Patient Christine Moretti Talks Positivity After a Diagnosis

PLS Patient Christine Moretti Talks Positivity After a Diagnosis

PLS Patient Christine Moretti Explains Why She Thought She Had ALS

PLS Patient Christine Moretti Explains Why She Thought She Had ALS

Genetic Counselors and Fabry Disease

Genetic Counselors and Fabry Disease

Fabry Disease - What a Clinician Needs to Know

Fabry Disease - What a Clinician Needs to Know

Newborn Screening and Fabry Disease: What We Know and Don't Know

Newborn Screening and Fabry Disease: What We Know and Don't Know

Tips for Clinicians and Dietitians Managing Late to Follow-up PKU Patients

Tips for Clinicians and Dietitians Managing Late to Follow-up PKU Patients

Sleepless Nights - Raising a Child with Sanfilippo Disease

Sleepless Nights - Raising a Child with Sanfilippo Disease

CEO of Albireo Pharmaceuticals Discusses the Company's Creation

CEO of Albireo Pharmaceuticals Discusses the Company's Creation

Ron Cooper Talks PFIC and A4250

Ron Cooper Talks PFIC and A4250

Daniel DeAngelo, MD, PhD Discusses Elderly AML Patients

Daniel DeAngelo, MD, PhD Discusses Elderly AML Patients

Daniel DeAngelo, MD, PhD Discusses AML Studies

Daniel DeAngelo, MD, PhD Discusses AML Studies

Daniel DeAngelo, MD, PhD Discusses His Study of GMI-1271 in Elderly AML Patients

Daniel DeAngelo, MD, PhD Discusses His Study of GMI-1271 in Elderly AML Patients

Daniel DeAngelo, MD, PhD Explains GMI-1271

Daniel DeAngelo, MD, PhD Explains GMI-1271

Daniel DeAngelo, MD, PhD Discusses Acute Myeloid Leukemia (Full Interview)

Daniel DeAngelo, MD, PhD Discusses Acute Myeloid Leukemia (Full Interview)

Gerhard Zugmaier, MD Discusses Acute Lymphoblastic Leukemia

Gerhard Zugmaier, MD Discusses Acute Lymphoblastic Leukemia

Ruben Mesa, MD Discusses His Phase 3 Study

Ruben Mesa, MD Discusses His Phase 3 Study

Ruben Mesa, MD Discusses Supportive Care for Myelofibrosis Patients

Ruben Mesa, MD Discusses Supportive Care for Myelofibrosis Patients

Ruben Mesa, MD Discusses Pathophysiology and Treatment of Myelofibrosis

Ruben Mesa, MD Discusses Pathophysiology and Treatment of Myelofibrosis

Ruben Mesa, MD on Myelofibrosis (Full Interview)

Ruben Mesa, MD on Myelofibrosis (Full Interview)

Serge Verstovsek, MD on Thrombocytosis

Serge Verstovsek, MD on Thrombocytosis

Serge Verstovsek, MD on Myelofibrosis

Serge Verstovsek, MD on Myelofibrosis