Lok Sabha Question No. 3375 (UNSTARRED), answered on 2019-07-12
Subject: Morquio A Syndrome
Ministry: HEALTH AND FAMILY WELFARE
Asked by: Parvesh Sahib Singh

## GOVERNMENT OF INDIA MINISTRY OF HEALTH AND FAMILY WELFARE DEPARTMENT OF HEALTH AND FAMILY WELFARE

## LOK SABHA UNSTARRED QUESTION NO. 3375 TO BE ANSWERED ON 12 TH JULY, 2019 MORQUIO A SYNDROME

## 3375. SHRI PARVESH SAHIBSINGH:

Will the Minister of HEALTH ANDFAMILY WELFARE be pleased to state:

- (a) whether the cure and treatment for Morquio A syndrome (MPS IV A) is available in India and if so, the details of types of treatment and places/clinics/hospitals where such treatment available;
- (b)  if  not,  whe ther  the  Government plans  to  bring  the  cure  by  international agreements /MoUs/Inter - Governmental cooperation  with  other  countries  like  USA and  if  so,  the  details thereof;
- (c) whether the cure and treatment for Morquio A syndrome (MPS IV A) is too costly t o be affordable by all people in India and if so, the details thereof indicating the average cost for the treatment of the above mentioned disorder in India; and
- (d) whether the Government proposes any financial assistance for the patients of rare genetic disorder like Morquio Asyndrome (MPS IV A) and if so, the details thereof?

## ANSWER THE MINISTER OF STATE IN THE MINISTRY OF HEALTH AND FAMILY WELFARE (SHRI ASHWINI KUMAR CHOUBEY)

(a)  to (d): Presently there is no cure available for Morquio A Syndrome (MPS IV A).  The currently  available  treatment  for  MPS  IV  A  is  Enzyme  Replacement  Therapy  (ERT).  The treatment is in the form of weekly intra - venous infusions of the ERT that can be administered at any secondary or tertiary care hospital.

Government had formulated a National Policy for Treatment of Rare Diseases in the y 2017. However, the Government decided to review the Policy in the light of new informa and  updates  available/received  for  its  further  improvement  and  effective impl mentation. Accordingly, the Policy has been kept in abeyance vide non - statutory Gazette Notification dated 18- 12- 2018, till the revised Policy is issued or until further orders, whichever is earlie

Pending revision of the National Po licy, under the Umbrella Scheme of Rashtriya Arogya Nidhi,  a  component  for  treatment  of  rare  disease  has  been  included  w.e.f.  01.01.201 providing one - time financial assistance to patients belonging to families living below thr poverty line for t reatment of identified Rare Diseases ( Annexure - I ) in Government hospitals. An amount of Rs.100 crore has been allocated for Rare Disease component of Umbrella Schem of Rashtriya Arogya Nidhi during 2019 - 20.

## List of identified rare diseases co vered for treatment under the Umbrella Scheme of Rashtriya Arogya Nidhi

## 1. Disorders amenable to treatment with Hematopoietic Stem Cell Transplantation (HSCT)

- i.Lysosomal storage disorders in early stages for which Enzyme replacement Therapy (ERT)  is  presently  not  available  (eg  Metachromatic  Leukodystrophy,  Krabbe's disease  and  severe  form  of  Mucopolysaccharoidosis  (MPS)  type  I  within  first years of age.
- ii.Adrenoleukodystr ophy (early stages), before the onset of hard neurological signs.
- iii. Immune  deficiency  disorders  eg  Severe  Combined  Immunodeficiency  (SCID), Chronic Granulomatous disease, Wiskot Aldrich Syndrome,etc
- iv.Osteopetrosis
- v. Fanconi Anemia
- vi.Others to be decided on case to case basis

## 2 . Disorders amenable to organ transplantation

- i. Liver  Transplantation -Metabolic  Liver  diseases and  other  Inborn  Errors  of Metabolism (small molecule diseases)
- i. Tyrosinemia  (failure  to  respond  to  nitisinone  therapy  or  have  documente evi dence of malignant changes in hepatic tissue)
- ii. Glycogen storage disorders (GSD) I, III and IV due to poor metabolic contr multiple liver adenomas, or high risk for Hepatocellualr carcinoma or evide of substantial cirrhosis or liver dysfunction or pro gressive liver failure,
- iii. MSUD (Maple Syrup Urine Disease),
- iv. Urea cycle disorders,
- v. Organic acidemias,
- vi. Wilson's disease (Decompensated cirrhosis).
- vii. Bile acid synthetic defects (Decompensated cirrhosis)
- viii. CriglerNajjar Type 1,
- ix. Alpha 1 antitrypsin deficiency (Decompensated cirrhosis)
- x. Progressive familial Intrahepatic Cholestasis (PFIC)(Decompensated cirrhosis)
- xi. Others to be decided -case to case basis

## ii. Renal Transplantation -

- i. Fabry's disease,
- ii. Autosomal recessive Polycystic Kidney Disease (ARPKD),
- iii. Autosomal d ominant Polycystic Kidney Disease (ADPKD)
- iv. Others on case to case basis
- iii. Patients  requiring  combined  liver  and  kidney  transplants - Rarely  Methyl Malonic aciduria may require combined liver & Kidney transplant) etc